The NF Conference Program Book & Abstract Book
The 2026 NF Conference Program Book and Abstract Book are now available for download. The Program Book includes
the full agenda, speaker information, and session descriptions. The Abstract Book features submitted abstracts
from this year's researchers and presenters.
Download 2026 Abstract Book
Download 2026 Program Book
Agenda Highlights
The 2026 NF Conference will feature presentations from leading experts in NF research and clinical care,
alongside insights from pioneering scientists in related disciplines. Attendees can look forward to dynamic
discussions, collaborative networking, and interactive satellite sessions that drive innovation across the
field.
Themes for the 2026 NF Conference include:
- Developmental Origins and Cellular Drivers of NF Disease
- Altered Metabolism in NF
- Tumor Immunology and Immunotherapy: From Antigens to Adoptive Cell Therapy
- NextGen Translational Pipelines: From Models to Clinical Impact
- Pain
- Neurocognition
To preview the caliber of topics and groundbreaking work typically showcased each year, please explore the 2025 NF Conference Program Book as well as our 2025 NF Conference Highlights report.
The agenda below is preliminary and subject to change. Please check back as we get closer to the NF Conference for the most up-to-date schedule.
Agenda
25 June, 2026 03:00 pm
to
07:00 pm
Registration & Check In
Centennial Foyer
Information & Help Desk is also open at this time.
26 June, 2026 07:00 am
to
05:00 pm
Registration & Check In
Centennial Foyer
Information & Help Desk is also open at this time.
26 June, 2026 08:30 am
to
05:30 pm
Young Investigator Day (Closed Satellite Program)
Alexandra O'Donohue
University of Sydney
Dr Alexandra O’Donohue is an early career postgraduate researcher from the Bioengineering & Molecular Medicine laboratory at the Westmead Institute for Medical Research and the University of Sydney. Dr O'Donohue received her BMedSci(Hons) in molecular biology and genetics in 2018 and then went on to complete her PhD from the University of Sydney in 2023. Throughout her postgraduate studies, she cultivated a keen interest in gene therapy, developing an expertise in CRISPR editing and the design, generation, and screening of tissue-specific recombinant adeno- associated vectors. Through mentorship with A/Prof Aaron Schindeler, she has cultivated her research interested in Neurofibromatosis type 1 and NF2-related Schwannomatosis. She has continued a body of work examining the links between metabolic changes associated with NF1 muscle weakness in bone, as well as expanded her gene therapy efforts for NF2-related Schwannomatosis. In 2025 she was awarded a fellowship via the US Children’s Tumor Foundation Young Investigator Award to support her research program.
Daochun Sun
Medical College of Wisconsin
Dr. Sun obtained his PhD degree in Molecular Biology and Genetics by studying mechanisms of Neurofibromatosis Type 1 (NF1)-associated Malignant Peripheral Nerve Sheath Tumors (MPNST) at School of Medicine, Wayne State University. He further received postdoctoral training from the Developmental Biology Department at the University of Texas, Southwestern Medical Center, and Cancer Biology and Genetics program in Memorial Sloan Kettering Cancer Center. His works emphasize the cell-of-origins of the tumor, and he identified a stem-like cell population playing essential roles in tumorigenesis, relapse, and metastasis of NF1-associated plexiform neurofibromas and MPNST. These discoveries may provide novel strategies to prevent tumor transformation, progression, chemoresistance, and metastasis.
Eduard Serra-Arenas
Germans Trias i Pujol Research Institute (IGTP)
Dr. Eduard Serra leads the Hereditary Cancer Group at the Germans Trias i Pujol Research Institute (IGTP), in Badalona (Barcelona), Spain. His group is part of the Cancer Translational Research Program (CARE) at IGTP. Research in his laboratory focuses on the study of tumors associated with Neurofibromatosis Type 1 (NF1), particularly those that arise in the peripheral nervous system. The group studies their development, composition, progression and possible therapies and patient management. The levels of study are the genomic analysis of tumors and the generation and the in vitro and in vivo use of cellular models, including 3D models derived from induced pluripotent stem cells (iPSCs). He is a close collaborator of different local and international associations of patients with NF1.
Larry Sherman
Oregon Health and Sciences University
Dr. Larry S. Sherman is the Assistant Chief and a Professor in the Division of Neuroscience at the Oregon National Primate Research Center and in the Neuroscience Graduate Program at the Oregon Health & Science University (OHSU). He is also the President of the Oregon and Southwest Washington Chapter of the Society for Neuroscience. He has over 120 publications related to brain development and neurodegenerative diseases including studies on neurofibromatosis and schwannomatosis. The Oregon Museum of Science and Industry and Portland Monthly Magazine recognized Dr. Sherman as one of the most innovative people in the State of Oregon. In 2012, he was recognized by the OHSU School of Medicine Faculty Senate and the OHSU foundation for Outstanding Teaching, and was awarded the Mary Omberg Award for outstanding support of science education in Oregon and Southwest Washington.
Rosalie Ferner
Guys and St Thomas' Hospital NHS Trust
Rosalie Ferner is an adult neurologist at Guy’s and St. Thomas’s NHS Foundation Trust. She was national lead clinician for the nationally commissioned Complex Neurofibromatosis 1 (NF1) Service from the launch in 2009 until 2022; she was the London lead for the national NF2 service from 2010-2014. She is a Trustee and member of the Medical Advisory Board for the patient organisation Nerve Tumours UK. She is a recipient of the European Theodor Schwann award (2016) and the von Recklinghausen award (2024).
She has extensive clinical and research experience in neurofibromatosis and schwannomatosis. The Neurofibromatosis Centre at Guy’s Hospital has about 2,000 patients and both children and adults are seen in the same department. The aim of the national NF1 service is to provide expert, lifelong, multi-disciplinary, holistic care for people with complex, multi-system disease. A transition service has been developed and expanded since 2009.
Rosalie Ferner’s doctoral thesis was on “Intellectual problems in neurofibromatosis 1” and her research interests include defining clinical phenotype and natural history in NF1, diagnosis of malignant peripheral nerve sheath tumours in NF1, and the development of robust clinical and patient focused outcome measures for monitoring therapy in NF1 and NF2 schwannomatosis.
Disclosures
Rosalie Ferner has acted as medical adviser on neurofibromatosis 1 for Astra Zeneca and Alexion and participated in educational sessions
Andrea McClatchey
Massachusetts General Hospital
Andrea “Andi” McClatchey is the Poitras Family Professor of Oncology at Massachusetts General Hospital and Harvard Medical School, and was named the 2011 Scott and Patricia Eston MGH ECOR Research Scholar in 2011. Dr. McClatchey received her PhD in Genetics from Harvard Medical School and completed postdoctoral training in the laboratory of Tyler Jacks at the Massachusetts Institute of Technology before joining the faculty at MGH and Harvard Medical School. In addition to running a research program, Dr. McClatchey is co-leader of the Landry Cancer Biology Consortium for graduate students at Harvard Medical School and devotes considerable time to the training and mentoring of graduate students. Dr. McClatchey’s research is dedicated to understanding the molecular basis of the familial tumor syndrome neurofibromatosis type 2 (NF2) and using that insight to develop and test new treatments for NF2. Her work has uncovered key aspects of how the NF2 protein Merlin functions and revealed fundamental rules by which all cells organize their outer membrane so as to appropriately interface with their environment. She actively engages in translating these fundamental scientific discoveries toward new therapeutic strategies for NF2 patients.
Centennial F
Session Chair: Vidya Browder, PhD, Children's Tumor Foundation
7 AM - 8:30 AM: Breakfast
8:30 AM - 8:35 AM: Welcome & Introduction by CTF
Vidya Browder, PhD, Children’s Tumor Foundation
8:35 AM - 8:50 AM: Introduction by all mentors
Andrea McClatchey, PhD, Massachusetts General Hospital, USA
Eduard Serra Arenas, PhD, Germans Trias i Pujol Research Institute, Spain
Helen Morrison, PhD, Leibniz Institute on Aging - Fritz Lipmann Institute, Germany
Vanessa Merker, PhD, Massachusetts General Hospital, USA
Daochun Sun, PhD, Medical College of Wisconsin, USA
Lawrence Sherman, PhD, Oregon Health and Science University, USA
Meena Upadhyaya, PhD, Cardiff University, UK
Rosalie Ferner, MD, Guys and St Thomas' Hospital NHS Trust, UK
8:50 AM - 9:05 AM: Icebreaker Activity
Vanessa Merker, PhD, Massachusetts General Hospital
9:05 AM - 10:05 AM: Platform Presentations
CRISPR-based editing for correcting NF2 pathogenic variants
Alexandra O'Donohue, Postdoctoral researcher, The University of Sydney
Schwann cell-specific loss of LZTR1 impairs lipid metabolism and activates ERK signalling, driving neuropathic pain and nerve hypertrophy
Georgia Daraki, PhD student, Fritz Lipmann Institute-Leibniz Institute on Aging
Preclinical modeling of differential targeting effects of CDK inhibitors in NF1-associated MPNST
Colin Beach, PhD student, Indiana University School of Medicine
Cell-Free DNA Testing for Early Detection of Malignant Peripheral Nerve Sheath Tumors among Adults with Neurofibromatosis Type 1: An International Delphi Consensus Study
Ramesh Lamsal, Postdoctoral researcher, Harvard Pilgrim Health Care Institute
10:05 AM - 10:15 AM: People's Choice Vote & Break
10:15 AM - 11:20 AM: Build a Career in NF Research: Lessons, Opportunities, and Future Directions
10:15 AM - 10:45 AM: Part I: Why I Chose NF Research and Why I Stayed (individual talks)
Rosalie Ferner, MD, Guys and St Thomas' Hospital NHS Trust
Meena Upadhyaya, PhD, Cardiff University
Larry Sherman, PhD, Oregon Health and Sciences University
10:45 AM - 11:20 AM: Part II: Your Questions, Their Experience: A Career Development Forum (panel discussion)
All mentors
11:20 AM - 12:20 PM: Elevator Pitch Workshop - Learn how to clearly and confidently communicate your research to different audiences in under two minutes
All mentors
12:20 AM - 1:15 PM: Lunch
1:15 PM - 2:45 PM: Funding Your Future - Grant writing strategies, grantee experiences, and small-group discussions
Andrea McClatchey, PhD, Massachusetts General Hospital
2:45 PM - 3:00 PM: Break
3:00 PM – 5:00 PM: Poster session
5:00 PM - 5:30 PM: Closing Remarks, People's Choice Award, & Feedback
All mentors
26 June, 2026 09:00 am
to
12:15 pm
REiNS Summer Meeting (Optional Satellite Meeting)
Centennial GH
Open to all patient advocates and clinician researchers
Email David Bonilla (dbonilla2@mgh.harvard.edu) for personalized registration link. Registration is requested for both in-person and virtual attendees to assist with space planning.
The REiNS (Response Evaluation in Neurofibromatosis Schwannomatosis) International Collaboration is a volunteer group of clinicians, researchers, and patients/family members who work together to improve the design of NF and SWN clinical trials. This meeting is open to any patients, advocates, clinicians, and researchers who would like to participate.
9:00 AM – 9:15 AM: Introduction and REiNS Leadership Update
Scott Plotkin, MD, PhD, Mass General Hospital
Brigitte Widemann, MD, National Cancer Institute
9:15 AM – 9:30 AM: Update from cutaneous neurofibroma working group: Patient-reported and clinician-reported outcomes
Laura Fertitta, MD, Henri Mondor University Hospital, Paris
9:30 AM – 9:45 AM: Update from functional outcomes working group: Decentralized Assessment of Hearing Outcomes in NF2-SWN
Andrea Gross, MD, Cincinnati Children's Hospital
9:45 AM – 10:00 AM: Update from biomarkers working group: Biomarkers for NF1 and SWN
R. Taylor Sundby, MD, Nemours Children's Health
10:00 AM – 10:15 AM: Update from imaging outcomes working group: Real-world experience with volumetry for plexiform neurofibroma
Eva Dombi, MD, National Cancer Institute
10:15 AM – 10:30 AM: Update from neurocognitive working group: Computerized battery of neurocognitive assessments in NF1/SWN
Jennifer Janusz, Psy.D., University of Colorado Anschutz
10:30 AM – 10:45 AM: Update from gene therapy working group: How perception of disease severity influences patient preferences for gene-targeted therapy
Miranda McManus, M.S., College of Charleston
10:45 AM – 11:00 AM: Update from patient-reported outcomes working group: Recommendations for the Assessment of Depression and Anxiety in Neurofibromatosis Clinical Trials
Heather Thompson, PhD, CCC-SLP, California State University, Sacramento
11:00 AM – 12:00 PM: REiNS Mini-Symposium: Patient/caregiver structured review of clinical trials for NF1/SWN
26 June, 2026 11:00 am
to
06:30 pm
NF Startup and Innovation Workshop (Closed Satellite Meeting)
Granite
Workshop Chair: Lydia The, PhD, Board of Directors, Children's Tumor Foundation
This is a closed workshop for a curated group selected from a call for applications.
26 June, 2026 01:00 pm
to
05:00 pm
Clinical Care Program, Day One - CME (Satellite Program, Registration Required)
Jonathan Payne
Murdoch Children’s Research Institute
Dr Payne is a neuropsychologist and clinician-scientist focused on neurodevelopmental outcomes in individuals with NF1. Cognitive, learning, and behavioral difficulties affect the majority of children with NF1 and are among the issues families consistently rank as having the greatest impact on daily life, yet treatment options remain limited. His work aims to change that. He co-leads the Brain & Mind group at the Murdoch Children's Research Institute, is a Senior Neuropsychologist at the Royal Children's Hospital, and is an Honorary Professorial Fellow at the University of Melbourne.
His research program integrates deep behavioral characterization with advanced neuroimaging and preclinical modelling to clarify our understanding of pathways linking genes, molecular signaling, brain, and behavior. He chairs the Medical Advisory Panel for CTF Australia, co-chairs the Neurocognitive Committee of the NF Clinical Trials Consortium, and chairs the Australian Paediatric Neuropsychology Research Network.
Karin S. Walsh
Children’s National Hospital
Karin S. Walsh, Psy.D. is a pediatric neuropsychologist at Children’s National Hospital and Professor of Pediatrics and Psychiatry & Behavioral Medicine at the George Washington University School of Medicine and Health Sciences. A clinician-scientist with over 20 years of experience, Dr. Walsh focuses on neurocognitive outcomes in children with neurofibromatosis type 1 (NF1), other RASopathies, pediatric brain tumors, and hematologic disorders. Her research examines executive function and cerebellar–cortical networks and leads cognitive intervention studies targeting learning and executive dysfunction in pediatric brain tumor and NF1 populations. Dr. Walsh has received multiple competitive research grants and serves in leadership roles across international consortia, including past president of the Posterior Fossa Society and current chair of the Neurocognitive Committee of the NF Clinical Trials Consortium.
Laura Klesse
UT Southwestern
Dr Laura Klesse is a pediatric neuro-oncologist who specializes in the care of patients with neurofibromatosis and central nervous system tumors. Dr. Klesse is currently the Director of the Comprehensive Neurofibromatosis and Associate Division Chief of Research Operations at UT Southwestern and Children’s Health in Dallas, Texas. She serves as the site’s principal investigator for the National NF Clinical Trials Consortium, the Children’s Oncology Group and as chair of the bone subcommittee for the NFCTC. Dr Klesse is nationally recognized as a clinical leader in NF patient care and is currently Chair of the Children’s Tumor Foundation’s Clinical Care Advisory Board. Dr Klesse is an Professor of Pediatrics and Neurological Surgery at UT Southwestern and is a Dedman Family Scholar in Clinical Care.
Tena Rosser
Children’s Hospital of Los Angeles
Dr. Tena Rosser is an Associate Professor of Pediatrics and Neurology at the Children’s Hospital Los Angeles which is affiliated with the USC Keck School of Medicine. She is the director of the CHLA Children’s Tumor Foundation-endorsed Neurofibromatosis Clinic which opened in 2005. She is also a member of the Children’s Tumor Foundation’s Clinical Care Advisory Board and the 2015 recipient of CTF Humanitarian Award. Dr. Rosser serves as the Los Angeles site Principal Investigator for the U.S. Army Department of Defense NF Consortium which coordinates multi-center clinical trials for individuals with NF1 and Schwannomatosis. She is a member of the DOD NF Consortium Quality of Life and Neurocognitive Committees. She cares for many children and adults with both NF1 and Schwannomatosis. She is a collaborator on translational NF1 and Schwannomatosis research projects with researchers across the country.
Alicia Gomes
University of Alabama at Birmingham
Alicia Gomes is a licensed and certified genetic counselor and earned her Master of Science in Genetic Counseling from the University of South Carolina in Columbia. Alicia served as laboratory genetic counselor for the University of Alabama at Birmingham (UAB) Medical Genomics Laboratory since 2010 within the UAB Department of Genetics. In 2022, Alicia transitioned to assistant professor at the University of Alabama at Birmingham and currently serves as the program director of the UAB Industry Genetics and Genomics Graduate Certificate Program and assistant director for the UAB Genetic Counseling Program. She also serves as coordinator for the NF-SWN Variant Curation Expert Panel, Vice-Chair of the Alabama Board of Genetic Counseling, and is an active member of the National Society of Genetic Counselors.
Amir Taghinia
Boston Children's Hospital
Amir Taghinia, MD, MPH, MBA is a plastic surgeon and hand surgeon at Boston Children's Hospital and Associate Professor of Surgery at Harvard Medical School. His academic and clinical interests include congenital and vascular anomalies, nerve conditions, and microsurgery. He has been treating patients with neurofibromatosis for over 15 years.
Jennifer Janusz
Children’s Hospital Colorado
Dr. Janusz is a pediatric neuropsychologist at Children’s Hospital Colorado (CHCO) and a Professor of Pediatrics and Neurology at the University of Colorado School of Medicine. She serves as Program Director of the CHCO Neurofibromatosis Program. Dr. Janusz is a member of the Children’s Tumor Foundation Clinical Care Advisory Board and chairs the Neurocognitive Committee of the REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) International Collaboration. Her research focuses on the neuropsychological and developmental profiles of children with a range of genetic and chromosomal disorders including neurofibromatosis, 22q11.2 deletion syndrome, and X and Y chromosome variants. Her research interests include executive functioning, learning, language development, and early cognitive trajectories, with the goal of translating findings into effective interventions. Dr. Janusz has secured grant funding and authored numerous publications in these areas.
Taylor Sundby
Nemours Children's Hospital
After completing undergraduate studies at Haverford College, Dr. R. Taylor Sundby received his M.D. from the Vanderbilt University School of Medicine in 2014. This was followed by his pediatric internship and residency training at the University of California San Francisco. Dr. Sundby joined the combined Pediatric Hematology and Oncology Fellowship training program at the National Cancer Institute (NCI), Pediatric Oncology Branch (POB) and Johns Hopkins University in 2017. In his fellowship, Dr. Sundby’s research focused on cancer predisposition genomics and developing non-invasive techniques for cancer surveillance. His fellowship research “Cell-free DNA ultra-low-pass whole genome sequencing to distinguish malignant peripheral nerve sheath tumor (MPNST) from its benign precursor lesion: A cross-sectional study” was selected as a 2022 NCI Center for Cancer Research top publication. Dr. Sundby became an Assistant Research Physician in 2022. He is a Francis S. Collins Scholar, past recipient of the Children’s Cancer Foundation NextGen Award and is board-certified in pediatrics and pediatric hematology/oncology.
Tina Jimenez
USCF Health
Dr. Tina Jimenez is a clinical neuropsychologist specializing in neurocognitive rehabilitation. She is an Assistant Professor in the Department of Neurological Surgery at UCSF, providing services in the Neurocognitive Care Clinic, an embedded clinic within the UCSF Brain Tumor Center. Her work focuses on advancing brain health, equity, and wellness among patients with a history of brain tumors and neurofibromatosis, with an emphasis on neurocognitive outcomes, quality of life, and survivorship. Dr. Jimenez serves on the Responsive Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration Neurocognitive Committee and the American Congress of Rehabilitation Medicine Oncology Cognitive Rehabilitation Task Force.
Centennial GH
Registration for the Clinical Care Program is required, and is separate from NF Conference registration.*
Session Co-Chairs:
Laura Klesse, MD, PhD, UT Southwestern
Tena Rosser, MD, Children's Hospital of Los Angeles
Nicole Ullrich, MD, PhD, Boston Children’s Hospital
1:00 PM - 1:15 PM: Clinical Care Program Welcome
Laura Klesse, MD, PhD, UT Southwestern
Tena Rosser, MD, Children's Hospital of Los Angeles
Nicole Ullrich, MD, PhD, Boston Children’s Hospital
1:15 PM - 1:45 PM: Invited Talk: A review of genotype-phenotype correlations in NF
Alicia Gomes, MS, CGC, University of Alabama at Birmingham
1:45 PM - 2:15 PM: Invited Talk: Surgical Management of Cutaneous and Peripheral Nerve Lesions in Neurofibromatosis Type 1 and Schwannomatosis
Amir Taghinia, MD, Boston Children’s Hospital
2:15 PM - 3:00 PM: Panel Discussion: Across Development: Neuropsychological Implications of NF1 from Infancy to Adulthood
Moderator:
Jennifer Janusz, PsyD, Children’s Hospital Colorado
Panel:
Jonathan Payne, DPsych, Murdoch Children's Research Institute
Nicole Ullrich, MD, PhD, Boston Children’s Hospital
Karin Walsh, PsyD, Children’s National Hospital
Tina Jimenez, PsyD, USCF Health
3:00 PM - 3:15 PM: Break
3:15 PM - 3:45 PM: Invited Talk: Closing the gap: Improving routine health surveillance for NF1 in primary care
Vanessa Merker, PhD, Massachusetts General Hospital & Harvard Medical School
3:45 PM - 4:00 PM: Day One Recap & Closing Remarks
Laura Klesse, MD, PhD, UT Southwestern
Tena Rosser, MD, Children's Hospital of Los Angeles
Nicole Ullrich, MD, PhD, Boston Children’s Hospital
4:00 PM - 5:00 PM: Clinical Care Program Networking Happy Hour - Denver Terrace, 5th Floor
Following the Day One session, Clinical Care Program attendees can enjoy cocktails, light refreshments, and networking on the Denver City Terrace. Attendance is strictly limited to registered Clinical Care Program participants.
26 June, 2026 02:00 pm
to
05:00 pm
CTF Board Meeting (Closed)
Silver
Closed meeting for CTF Board of Directors
27 June, 2026 07:00 am
to
05:00 pm
Registration & Check In
Centennial Foyer
Information & Help Desk is also open at this time.
27 June, 2026 07:00 am
to
08:00 am
Clinic Coordinators Breakfast Meeting (RSVP required)
Centennial F
27 June, 2026 08:00 am
to
11:45 am
Clinical Care Program, Day Two - CME (Satellite Program)
Laura Klesse
UT Southwestern
Dr Laura Klesse is a pediatric neuro-oncologist who specializes in the care of patients with neurofibromatosis and central nervous system tumors. Dr. Klesse is currently the Director of the Comprehensive Neurofibromatosis and Associate Division Chief of Research Operations at UT Southwestern and Children’s Health in Dallas, Texas. She serves as the site’s principal investigator for the National NF Clinical Trials Consortium, the Children’s Oncology Group and as chair of the bone subcommittee for the NFCTC. Dr Klesse is nationally recognized as a clinical leader in NF patient care and is currently Chair of the Children’s Tumor Foundation’s Clinical Care Advisory Board. Dr Klesse is an Professor of Pediatrics and Neurological Surgery at UT Southwestern and is a Dedman Family Scholar in Clinical Care.
Andrea Gross
Cincinnatti Children's Hospital Medical Center
Dr. Gross is a pediatric oncologist who focuses on clinical trials research and tumor predisposition syndromes, such as neurofibromatosis type 1 (NF1). Her areas of interest include developing and utilizing functional outcome measures for tumor predisposition syndromes, working with rare disease patient advocates to increase patient engagement in clinical trial design and dealing with the challenge of medication adherence in the NF1 population.
Carlos Romo
Johns Hopkins Medical Institute
Dr. Carlos Romo is a neuro-oncologist who specializes in the evaluation and treatment of people with neurofibromatosis. Dr. Romo earned his medical degree from the School of Medicine and Health Sciences at Tecnológico de Monterrey in Mexico and completed his Neurology residency at the University of Arkansas for Medical Sciences. He then completed a Neuro-oncology clinical and research fellowship in a joint program between The Johns Hopkins University and the National Institutes of Health. Dr. Romo additionally trained as a clinical pharmacology fellow at The Johns Hopkins University and completed training on methods for clinical research at the University of Texas MD Anderson Cancer Center. In his research, Dr. Romo studies the natural history of cutaneous and plexiform neurofibromas, as well as new and more effective ways to monitor and treat neurofibromas and tumors of the central nervous system.
Clement Cheung
Children's Hospital Los Angels
Dr. Clement Cheung is a Clinical Associate Professor of Pediatrics at the Keck School of Medicine of the University of Southern California. He received his MD and PhD from the University of Washington, where his doctoral research focused on reproductive neuroendocrinology, including leptin signaling as a nutritional cue for pubertal onset in rodent models. He completed pediatric residency and pediatric endocrinology fellowship training at the University of California, San Francisco, and subsequently conducted NIH K08-funded research on the molecular biology of hypothalamic control of energy homeostasis. He joined Children’s Hospital Los Angeles in 2012 and is Director of the Neuroendocrinology Clinic, providing endocrine care for children with brain tumors, pituitary disorders, and other conditions affecting hypothalamic and pituitary function. He also serves as Associate Program Director for the Pediatric Endocrinology Fellowship and the Pediatric Residency Program, and he previously served as the inaugural Director of Endocrine Medical Education for residents and medical students. He is committed to medical education and mentorship, supporting trainees and learners in clinical scholarship and research. He is completing a Master of Academic Medicine at USC Keck School of Medicine (expected 2026).
John Binder
Children's Hospital Colorado
Dr. John Binder is an Associate Professor of Child Neurology and Co-Director of the Neurofibromatosis Program at Children's Hospital Colorado. He specializes in the neurologic care of patients with neurofibromatosis and participates in both dedicated neurology NF clinics and multidisciplinary NF care. In addition to his work in neurofibromatosis, Dr. Binder maintains a broad outpatient pediatric neurology practice and is actively involved in resident education and teaching. He values collaborative, team-based care and enjoys working closely with colleagues across specialties to support children and families.
Justin Jordan
UT Southwestern Medical Center
Dr. Jordan is Associate Professor of Neurology with a secondary appointment in the Peter O’Donnell Jr. Brain Institute at the University of Texas Southwestern Medical Center in Dallas, Texas. He serves as Head of the Neuro-Oncology Section, where he also runs a comprehensive clinic for adult patients affected by NF1 and schwannomatosis. Dr. Jordan is also involved in a variety of research topics including clinical trials for cutaneous neurofibromas, NF1-associated gliomas, NF2-related tumors, and chronic pain in non-NF2-related schwannomatosis.
Kate Kelts
Children's Tumor Foundation
Kate Kelts is a registered nurse and the Vice President of Research, Impact, and Engagement at the Children's Tumor Foundation (CTF). With extensive experience as a pediatric nurse, Kate spent many years providing care to acutely ill children in hospital settings before transitioning to a role as Clinic Coordinator in the Neurology Department at Children’s Hospital of Los Angeles (CHLA). It was there that she first encountered families affected by Neurofibromatosis (NF) Type 1, NF2-related schwannomatosis, and Schwannomatosis. Driven by a passion for supporting patients and their families, Kate joined CTF in 2015 as a Patient Support Coordinator. Since then, her role has evolved significantly, but her commitment to providing education, support, and advocacy for families living with NF has remained central to her work. Kate oversees key initiatives such as the NF Conference, the premier global meeting for NF research and clinical care, and the NF Summit, the largest annual gathering of patients and families affected by NF. She also leads CTF Engage, a patient representative training program that empowers individuals living with NF to actively engage in research by prioritizing their voices and lived experiences. Additionally, Kate serves as Principal Investigator for the NF Registry, the world's largest patient-reported database for individuals with any form of NF, and works closely with CTF’s Clinical Care Advisory Board to improve the quality of care for patients and families living with NF. Kate’s dedication to advancing patient education, support, and research in the NF community continues to drive her work at the Children’s Tumor Foundation, where she remains focused on making a meaningful impact in the lives of those affected by these rare genetic conditions.
Nicole Ullrich
Boston Children’s Hospital
Dr. Ullrich completed her medical and graduate degrees at Yale University followed by residency in Pediatrics and Neurology at Boston Children’s Hospital, where she then did her fellowship in NeuroOncology before joining the faculty in the Department of Neurology. Her research focuses on neurologic and oncologic complications of NF1 and the long-term neurologic complications of childhood systemic cancer and brain tumors. Dr. Ullrich has been involved in the design and execution of clinical trials for complications of NF through the Neurofibromatosis Clinical Trials Consortium, where she is currently the site principal investigator for the Harvard site that includes Boston Children’s Hospital, Dana-Farber Cancer Institute and Massachusetts General Hospital. Within the consortium, she is former chair of the Low Grade Glioma committee and currently co-chair of the Neurocognitive Committee. She serves as co-Chair of the Clinical Research Award committee and is a member of the Clinical Care Advisory Board for the Children’s Tumor Foundation. Lastly, she serves on the board of NF Northeast. Dr. Ullrich recently participated in the international working groups to revise and update the diagnostic criteria for the neurofibromatoses and in the updated AAP health supervision guidelines for children with NF1.
Centennial GH
*Registration for the Clinical Care Program is required, and is separate from NF Conference registration.*
Session Co-Chairs:
Laura Klesse, MD, PhD, UT Southwestern
Tena Rosser, MD, Children's Hospital of Los Angeles
Nicole Ullrich, MD, PhD, Boston Children’s Hospital
7:00 AM - 8:00 AM: Breakfast is provided to registered attendees of the Clinical Care Program at Centennial GH
8:00 AM - 8:10 AM: Welcome & Updates from the Clinical Care Advisory Board
Laura Klesse, MD, PhD, UT Southwestern
8:10 AM - 8:30 AM: Invited Talk: The NF Registry: Advancing Impact & Engagement
Kate Kelts, RN, BSN, Children’s Tumor Foundation
8:30 AM - 9:00 AM
Invited Talk: Too Early, Too Slow, or Too Much: Endocrine Clues You Can't Miss in Neurofibromatosis Type 1
Clement Cheung, MD, PhD, Children's Hospital Los Angeles
9:00 AM - 9:30 AM: Invited Talk: From Bench to Bedside: Biomarkers in NF1 Clinical Care
Taylor Sundby, MD, Nemours Children’s Health NF Clinic
9:30 AM - 10:00 AM: Invited Talk: Real World Application of Updated Schwannomatosis Diagnosis Criteria
Justin Jordan, MD, PhD, UT Southwestern
10:00 AM - 10:15 AM: Break
10:15 AM - 10:45 AM: Panel Discussion: Atypical Neurofibromas in NF1: Diagnostic Challenges and Malignant Potential
Moderator:
Laura Klesse, MD, PhD, UT Southwestern
Panel:
Andrea Gross, MD, Cincinnati Children's Hospital
Carlos Romo, MD, Johns Hopkins School of Medicine
10:45 AM - 11:30 AM: Panel Discussion: Transition of Care in NF1: A Tale of Two Patients
Moderators:
Angela Hirbe, MD, PhD, Washington University School of Medicine
Tena Rosser, MD, Children’s Hospital Los Angeles
Panel:
John Binder, MD, Children’s Hospital Colorado
Justin Jordan, MD, PhD, UT Southwestern
11:30 AM - 11:45 AM: Closing Remarks
Nicole Ullrich, MD, PhD, Boston Children’s Hospital
27 June, 2026 09:00 am
to
12:00 pm
Data Workshop (Satellite Session)
Daochun Sun
Medical College of Wisconsin
Dr. Sun obtained his PhD degree in Molecular Biology and Genetics by studying mechanisms of Neurofibromatosis Type 1 (NF1)-associated Malignant Peripheral Nerve Sheath Tumors (MPNST) at School of Medicine, Wayne State University. He further received postdoctoral training from the Developmental Biology Department at the University of Texas, Southwestern Medical Center, and Cancer Biology and Genetics program in Memorial Sloan Kettering Cancer Center. His works emphasize the cell-of-origins of the tumor, and he identified a stem-like cell population playing essential roles in tumorigenesis, relapse, and metastasis of NF1-associated plexiform neurofibromas and MPNST. These discoveries may provide novel strategies to prevent tumor transformation, progression, chemoresistance, and metastasis.
Robert Allaway PhD
Sage Bionetworks
Robert Allaway is a principal scientist at Sage Bionetworks. His research focuses on applying computational methods to study rare diseases like neurofibromatosis. In addition to his research role, he also leads projects with the broad goal of promoting the use of open science methods in biomedical research and algorithm development for biomedical applications.
Kimani Njoya
Medical College of Wisconsin
Kimani Njoya is a PhD candidate in the Department of Cell Biology, Neurobiology and Anatomy at the Medical College of Wisconsin in the lab of Dr. Daochun Sun. His research focuses on intercellular signaling and metabolic reprogramming in neurofibromatosis type 1 (NF1)-associated tumors. His scientific training spans experimental and computational approaches across genomics, transcriptomics, and metabolomics at bulk, single-cell, and spatial resolution. He is a recipient of the Children’s Tumor Foundation NF Data Utilization Award, supporting his work integrating lipidomics and transcriptomics to identify targetable metabolic adaptations in NF1 tumors. His first-author study, published in npj Precision Oncology (2025), investigates the natural history of SPP1 signaling across NF1 tumor initiation and progression, and he has co-authored peer-reviewed publications spanning therapeutic strategies for NF1 tumors, vector biology, and computational enhancer prediction.
Aditya Nath
Sage Bionetworks
Aditya Nath is a Biomedical Data manager at Sage Bionetworks, who works with the Rare disease team to facilitate uploading data to the NF portal, and then releasing that data for researchers to explore. He joined Sage about a year ago, having worked at the Allen institute for Cell Science previously.
Kate Kelts
Children's Tumor Foundation
Kate Kelts is a registered nurse and the Vice President of Research, Impact, and Engagement at the Children's Tumor Foundation (CTF). With extensive experience as a pediatric nurse, Kate spent many years providing care to acutely ill children in hospital settings before transitioning to a role as Clinic Coordinator in the Neurology Department at Children’s Hospital of Los Angeles (CHLA). It was there that she first encountered families affected by Neurofibromatosis (NF) Type 1, NF2-related schwannomatosis, and Schwannomatosis. Driven by a passion for supporting patients and their families, Kate joined CTF in 2015 as a Patient Support Coordinator. Since then, her role has evolved significantly, but her commitment to providing education, support, and advocacy for families living with NF has remained central to her work. Kate oversees key initiatives such as the NF Conference, the premier global meeting for NF research and clinical care, and the NF Summit, the largest annual gathering of patients and families affected by NF. She also leads CTF Engage, a patient representative training program that empowers individuals living with NF to actively engage in research by prioritizing their voices and lived experiences. Additionally, Kate serves as Principal Investigator for the NF Registry, the world's largest patient-reported database for individuals with any form of NF, and works closely with CTF’s Clinical Care Advisory Board to improve the quality of care for patients and families living with NF. Kate’s dedication to advancing patient education, support, and research in the NF community continues to drive her work at the Children’s Tumor Foundation, where she remains focused on making a meaningful impact in the lives of those affected by these rare genetic conditions.
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Workshop Chairs:
Aditya Nath, PhD, Sage Bionetworks
Kara Quaid, PhD, Children's Tumor Foundation
Belinda Garana, PhD, Sage Bionetworks
Robert Allaway, PhD, Sage Bionetworks
Irene Morganstern, PhD, Children's Tumor Foundation
9:00 AM - 9:15 AM: Keynote – Why data generation/re-use is important
Daochun Sun, PhD, Medical College of Wisconsin
9:15 AM - 9:45 AM: The NF Data Portal in Action: Analyzing Data & Accelerating Research
Aditya Nath, MS, Sage Bionetworks
Belinda Garana, PhD, Sage Bionetworks
Robert Allaway, PhD, Sage Bionetworks
Andrew Goodspeed, PhD, Pluto Biosciences
Mea Casey, PhD, Pluto Biosciences
9:45 AM - 10:45 AM: Lightning Talks
9:45 AM - 9:55 AM: The NF Registry: Advancing Impact & Engagement
Kate Kelts, RN, BSN, Children’s Tumor Foundation
9:55 AM - 10:05 AM: AI-Driven Drug Discovery for NF2-Related Schwannomatosis: Identifying Novel Therapeutic Candidates
Phillip Brownjohn, PhD, Healx AI
10:05 AM - 10:15 AM: Decoding metabolic reprogramming in NF1 tumors: From data-driven discovery to therapeutic insights
Kimani Njoya, Medical College of Wisconsin
10:15 AM - 10:25 AM: Multi-phenotype GWAS with AI-assisted phenotyping identifies novel loci associated with cutaneous neurofibroma development
Michelle Lin, Stanford University School of Medicine
10:25 AM - 10:35 AM: The NF Target Hub: Connecting Transcriptomic Data to Targets in Neurofibromatosis
Kara Quaid, PhD, Children’s Tumor Foundation
10:35 AM - 10:45 AM: Q&A
10:45 AM - 11:00 AM: Break
11:00 AM - 12:00 PM: Hands-On Workshop: Find and Analyze Data with the NF Data Portal
Kara Quaid, PhD, Children’s Tumor Foundation
Robert Allaway, PhD, Sage Bionetworks
Andrew Goodspeed, PhD, Pluto Biosciences
27 June, 2026 09:00 am
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05:00 pm
NF AI Innovation Workshop (Satellite Session)
Energy Ballroom at Homewood Suites by Hilton Denver Downtown 550 15th Street, Denver, Colorado, 80202
The NF AI Innovation Workshop is a dedicated session exploring the rapidly evolving role of artificial intelligence in neurofibromatosis and schwannomatosis research. The workshop focuses on how modern AI tools can accelerate scientific discovery, translational research, and clinical impact, and features two connected sessions:
Part 1: Discovery & Discussion Session (9:00 AM – 11:00 AM) Open to all attendees. This session includes a virtual presentation from Jonah Cool, followed by panel discussions with researchers, clinicians, and AI practitioners on emerging AI capabilities, practical applications in biomedical research, and new opportunities within the NF ecosystem.
Part 2: Closed Working Session (12:00 PM – 5:00 PM) Closed session; pre-application required. Following the morning presentations, accepted participants will engage in a focused, hands-on workshop. Researchers and teams will collaborate directly with AI experts, TAs, and expert coaches to build actionable proof-of-concept projects, experimental workflows, and research ideas using cutting-edge agentic AI tools.
Who Should Attend:
Researchers and clinicians interested in applying modern AI tools to NF research.
Participants looking to learn about emerging agentic AI capabilities.
Teams developing hands-on proof-of-concept projects and workflows.
Note: While the afternoon Working Session required a prior application, all conference attendees are welcome to attend and participate in the morning Discovery & Discussion Session. Breakfast and lunch will be served.
27 June, 2026 12:00 pm
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01:30 pm
Lunch
Centennial Foyer
Box lunches are available for purchase daily, Saturday through Tuesday. To save time and avoid on-site concession lines, please purchase your box lunch(es) during registration.
27 June, 2026 12:15 pm
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01:15 pm
NF Startup and Innovation Showcase (Satellite)
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Session Chair: Lydia The, PhD
Venture capital plays a critical role in translating promising therapeutic ideas into funded programs, particularly in advancing innovations through early clinical development. This session is designed to connect the venture community with the NF scientific ecosystem. It will be structured in two parts. First, a brief presentation from a venture investor on how funding decisions are made today, including current biotech trends and considerations specific to NF. Second, presentations from 3-4 teams working on promising companies or translational ideas, followed by a discussion with VC and biotech mentors. This session will provide a transparent view into how early-stage opportunities are evaluated, refined, and positioned for funding.
27 June, 2026 01:30 pm
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02:00 pm
Opening Remarks
Sylwia Ammoun
University of Plymouth
Dr. Sylwia Ammoun is currently a Senior Research Fellow at the Peninsula Medical School, University of Plymouth (UK). She earned her PhD in 2005 from Uppsala University, Sweden, where she researched the pharmacology and signalling mechanisms of orexin receptors.
In 2006, Dr. Ammoun joined Professor Oliver Hanemann’s laboratory at the University of Plymouth, UK. As a member of the Brain Tumour Research Centre of Excellence, she investigates the pathobiology of NF2-related and sporadic meningiomas and schwannomas, aiming to identify novel therapeutic targets and develop more effective treatments.
Her research as an independent researcher has demonstrated that receptors such as MERTK, AXL, PDGFRβ, ErbB2/3, and IGF-IR, along with Human Endogenous Retroviruses (HERVs) and cellular prion proteins, contribute to the pathobiology of schwannoma and meningioma tumours. She is currently investigating the role of the tumour microenvironment and approaches to enhance drug sensitivity. This work has led to key translational outcomes, including one in vivo study and two Phase 0 clinical trials in NF2-related schwannomatosis (NF2-SWN), where she served as co-investigator in one trial, principal investigator in the ongoing trial, and Director of Studies for the in vivo project.
Theresa Miller
Congressionally Directed Medical Research Programs
Dr. Theresa J. Miller currently serves as the Program Manager for the Kidney Cancer Research Program (KCRP) and the Neurofibromatosis Research Program (NFRP) for the Congressionally Directed Medical Research Programs, U.S. Army Medical Research and Development Command, Fort Detrick, Maryland.
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Welcome Remarks
Annette Bakker, PhD, Children's Tumor Foundation
Theresa Miller, PhD, Congressionally Directed Medical Research Programs
Opening Remarks from 2026 Conference Co-Chairs
Steven Rhodes, MD, PhD, Indiana University
David Largaespada, PhD, University of Minnesota
Sylwia Ammoun, PhD, University of Plymouth
27 June, 2026 02:00 pm
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03:00 pm
Keynote: Hyaluronan synthesis, catabolism and signaling as therapeutic targets for peripheral nerve tumors
Larry Sherman
Oregon Health and Sciences University
Dr. Larry S. Sherman is the Assistant Chief and a Professor in the Division of Neuroscience at the Oregon National Primate Research Center and in the Neuroscience Graduate Program at the Oregon Health & Science University (OHSU). He is also the President of the Oregon and Southwest Washington Chapter of the Society for Neuroscience. He has over 120 publications related to brain development and neurodegenerative diseases including studies on neurofibromatosis and schwannomatosis. The Oregon Museum of Science and Industry and Portland Monthly Magazine recognized Dr. Sherman as one of the most innovative people in the State of Oregon. In 2012, he was recognized by the OHSU School of Medicine Faculty Senate and the OHSU foundation for Outstanding Teaching, and was awarded the Mary Omberg Award for outstanding support of science education in Oregon and Southwest Washington.
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Larry Sherman, PhD, Oregon Health and Sciences University
27 June, 2026 03:00 pm
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05:00 pm
Developmental Origins and Cellular Drivers of NF Disease Session 1
Benjamin Callaway
Miami University
Benjamin J. Callaway is a PhD candidate in the Department of Biology at Miami University. studying CRISPR/Cas9 zebrafish models for Neurofibromatosis Type 1 (NF1). Their personal interests are in how germline modifiers influence immune-tumor interactions. They will present findings on a novel CRISPR-based zebrafish model of NF1 suitable for studying central nervous system tumors.
Claudia Barros
University of Plymouth
Dr Claudia Barros is an Associate Professor of Neuroscience at the Peninsula Medical School of the University of Plymouth (UoP), and a research lead at the UK Brain Tumour Research Centre of Excellence hosted by UoP. She investigates glioma development and targeting, with an interest also in neural stem cell (NSC) research. Dr Barros received her 5-year Diploma degree from the University of Lisbon, Portugal, her doctorate from Cambridge University UK, and was a postdoctoral fellow at both the Scripps Research Institute, La Jolla, USA and at the Wellcome Centre for Human Genetics, Oxford University, UK. She was awarded a Leverhulme Trust Early Career Fellowship before joining UoP. Her team takes advantage of the highly genetically tractable Drosophila brain as in vivo discovery platform and develops models using patient-derived glioma stem cells, low grade and high-grade glioma. Dr Barros’ team contributed to demonstrate that the Hippo pathway maintains NSC quiescence and revealed that a STRIPAK complex orchestrates Hippo and Insulin signalling to reactivate NSCs. More recently, her team exposed candidate signals responsible for the very initial stages of brain tumourigenesis and partnered with drug discovery companies addressing the action of their inhibitors in glioma in mono and combination therapies, taking their work another step forward in the discovery of new therapeutics.
David Parkinson
University of Plymouth
‘Professor David Parkinson is the Director of the Brain Tumour Research Centre at the University of Plymouth, UK. His undergraduate training was at King’s College, followed by a Medical Research Council funded PhD at University College London. Following a post-doctoral fellowship from Cancer Research UK at the London Research Institute, he then moved to University College London. In 2008, he moved to Plymouth as an independent PI. His work in Plymouth began with understanding mechanisms of PNS repair, before focussing on the biology of Merlin-null schwannoma and meningioma tumours. His group now uses human primary tumour cell models as well as pre-clinical mouse models to understand the biology of these tumours and trial new therapies.’
Elisabeth Castellanos
German Trias i Pujol Research Institute (IGTP)
Dr. Elisabeth Castellanos has led the Clinical Genomics Unit within the Genetics Service at Germans Trias i Pujol University Hospital since 2019. Her laboratory provides molecular diagnosis of inherited genetic disorders, with a strong focus on neurofibromatosis (NF) and schwannomatosis, among other rare genetic diseases. Since 2015, the Genetics Service at Germans Trias i Pujol Hospital has been designated as one of Spain’s two national Reference Centers (CSUR) for phakomatoses, reflecting its recognized clinical and diagnostic expertise. More recently, the group has joined GENTURIS, the European Reference Network for genetic tumor risk syndromes.
Alongside her clinical responsibilities, Dr. Castellanos leads a translational research program dedicated to improving the diagnosis and treatment of patients with neurofibromatosis and schwannomatosis. Her work has resulted in multiple scientific publications that advance genetic testing strategies, establish novel cellular models to investigate the role of NF–SWN genes in tumorigenesis, and explore innovative RNA-based therapeutic approaches for NF2-related Schwannomatosis.
Dr. Castellanos currently leads the EURONET-NF consortium, an international initiative aimed at developing and harmonizing advanced genetic diagnostic tools for neurofibromatosis and schwannomatosis. In addition, together with Dr. Scott Plotkin, she co-leads the ClinGen Variant Curation Expert Panel for Neurofibromatosis and Schwannomatosis (NF-SWN VCEP), which is responsible for defining international standards for the classification of variants in NF-SWN–associated genes.
Jonas Van Lent
Institute of Oncology Research (IOR),
Dr. Jonas Van Lent is an Independent Young Investigator at the Institute of Oncology Research (IOR), Switzerland. His research focuses on human pluripotent stem cell (hPSC)-based models of the peripheral nervous system to study tumor microenvironment dynamics, neuro-immune interactions, and neuro-oncology. He currently applies these approaches to Neurofibromatosis, investigating Schwann cell tumor biology and mechanisms of tumor progression.
He completed his PhD at the University of Antwerp (Belgium), where he developed stem cell-based models for inherited neuropathies, including Charcot-Marie-Tooth disease. His work provided insights into mitochondrial dysfunction, axonal degeneration, and Schwann cell myelination defects, and was recognized with awards including the P.K. Thomas Prize and selection in CMTA-STAR’s 40 Under 40. In 2025, he received the Children’s Tumor Foundation Young Investigator Award for his work on Neurofibromatosis.
His current work integrates advanced stem cell technologies to develop complex human PNS models, including assembloids and tumor-nerve-immune co-cultures, to study NF-associated tumor development and microenvironment interactions.
Judith Kempfle
UMass Chan Medical School/UMass Memorial Medical Center & Massachusetts Eye and Ear/Harvard Medical School
Dr. Judith Kempfle is an otologist and surgeon–scientist at UMass Chan Medical School/UMass Memorial Medical Center and Massachusetts Eye and Ear/Harvard Medical School, where she provides specialized care for patients with hearing and balance disorders, while leading a research program focused on inner ear regeneration and hearing restoration, with particular emphasis on Schwann cell biology, neurotrophin signaling, and immune–glial interactions in conditions such as sensorineural hearing loss and NF2-related schwannomatosis.
Khalil Ali Ahmad Kasm
University of Texas MD Anderson Cancer Center
Dr. Khalil A. Kasm is a postdoctoral fellow at The University of Texas MD Anderson Cancer Center studying cancer neuroscience and the role of sensory neurons in tumor progression and cancer pain. His research focuses on how TRPV1⁺ nociceptor activity promotes malignant peripheral nerve sheath tumor (MPNST) growth through neuron-tumor interactions. His work aims to identify novel therapeutic targets at the interface of neuroscience and oncology.
Maxwell Laws
National Institutes of Health
Maxwell T. Laws, MD, is a seventh-year neurosurgery resident in the NIH/University of Virginia Neurosurgery Program and a clinical/postdoctoral fellow in the Chittiboina Laboratory at the NIH. He earned his B.S. in Biomedical Engineering from Wayne State University, where he worked in the laboratory of Juri G. Gelovani, MD, PhD, developing and validating radioligands for epigenetic profiling of glioblastoma. He subsequently received his M.D. with Distinction from Wayne State University School of Medicine.
Dr. Laws’ research focuses on the epigenetic and immunologic basis of neurologic disease, with current areas of investigation including neurofibromatosis type 2, Von Hippel-Lindau syndrome, and anaplastic ependymoma. His primary work aims to define tumorigenic pathways in NF2-related central nervous system cancers. As part of this work, he has developed bioinformatic signatures, including a merlin depletion score, to characterize the spectrum of functional merlin loss in NF2-related tumors and identify associated proliferative, angiogenic, and immune programs. His translational efforts include investigating TEAD inhibitor therapy as a targeted strategy for merlin-deficient tumors, with the goal of moving mechanistic discoveries into the clinic.
Sajjad Khan
Medical College of Wisconsin
Dr. Sajjad Ali Khan is a translational cancer biologist and Postdoctoral Fellow at the Medical College of Wisconsin, where he investigates mechanisms of tumor heterogeneity and therapeutic resistance in NF1-associated tumors. He holds a PharmD and earned a PhD in Cell and Molecular Biology from the University of Strathclyde, UK, where his work focused on anticancer bioactive molecules and signaling pathways.
His research focuses on identifying therapeutic vulnerabilities that arise from developmentally distinct tumor cell states. Using integrated single-cell transcriptomic, functional, and in vivo approaches, he investigates how stem-like tumor populations drive disease progression and treatment response in plexiform neurofibroma (PN) and malignant peripheral nerve sheath tumors (MPNST). His work supports a model in which a limited population of stem-like cells with distinct signaling dependencies sustains tumor growth, drives therapy resistance, and contributes to recurrence. He has identified key signaling dependencies that sustain these stem-like tumor states and leverages this biology to design combinatorial therapeutic strategies. During his postdoctoral training, he has led multiple first-author studies in NF1-associated tumors. More recently, his research has expanded to explore metabolic dependencies of stem-like tumor cells, with the goal of uncovering additional vulnerabilities for therapeutic intervention.
Prior to joining MCW, Dr. Khan served as an Assistant Professor of Pharmacology, where he led a research program in drug discovery and disease pathophysiology, including studies in nephrotoxicity and hepatotoxicity. He supervised pharmacy and graduate students on projects in target validation and cell signaling, contributing to multiple peer-reviewed publications. His work spans mechanistic cancer biology, preclinical therapeutics, and translational strategy, with a focus on identifying actionable vulnerabilities that can be leveraged for improved patient outcomes.
Yuan Zhu
UT Southwestern Medical Center
Dr. Yuan Zhu is a Professor in the Department of Pediatrics and Harold C. Simmons Comprehensive Cancer Center at UT Southwestern (UTSW) Medical Center at Dallas. Dr. Zhu holds Children’s Cancer Fund Distinguished Professorship in Pediatric Oncology Research.
Dr. Zhu received a Bachelor of Science degree in Biochemistry from Fudan University and obtained his Ph.D. in Neuroscience from UTSW in 2000. He was a tenure-track Assistant Professor (2003 to 2010), and an Associate Professor with tenure (2010 to 2013) in the Departments of Internal Medicine and Cell & Developmental Biology at the University of Michigan Medical School. In 2013, Dr. Zhu joined Children’s National Hospital in Washington D.C. where he served as Scientific Director of the Gilbert Family Neurofibromatosis (NF) Institute and Associate Director of Research in the Center for Cancer and Immunology Research.
Dr. Zhu’s research findings in cancer research, particularly in the fields of brain tumors and NF1, have been published in highly selective journals, including Science, Cell, Nature Reviews Cancer, Cancer Cell, Nature Communications, and Developmental Cell. His work has identified the mechanisms by which alterations in the NF1 tumor suppressor gene (TSG) and other TSGs (e.g., p53) contribute to developmental alterations and tumorigenesis in the nervous system. Dr. Zhu’s work has contributed to the understanding of pathogenesis and clinical behaviors of tumors arising in individuals with NF1 as well as in a range of common cancer types where NF1 gene alterations arise somatically and consequently abnormally activate RAS-mediated ERK/MAPK signaling during cancer development. His recent work has led to a Clinical Trial Award of the Department of Defense for NF1-associated malignant peripheral nerve sheath tumors and atypical neurofibromas. This clinical trial was activated on April 1, 2025.
Dr. Zhu received academic and research awards, including the University of Michigan Biological Sciences Scholar Program Scholar and American Cancer Society Research Scholar, and the Gilbert Family Endowed Chair in Neurofibromatosis Research. Dr. Zhu was elected to a fellow by the American Association for the Advancement of Science (AAAS) in 2019. Dr. Zhu received the inaugural Outstanding Scientist Award from the George Washington University Cancer Center in 2021 and was selected as a Cancer Prevention and Research Institute of Texas (CPRIT) Scholar in Cancer Research for Established Investigators in 2023.
Niveditha Ravindra
National Institutes of Health
Nivi Ravindra, MD, FACMG is a pathologist, board-certified laboratory geneticist and post-
doctoral fellow at the National Institute of Neurological Disease and Stroke, NIH, where her
research focuses on the genomic basis and mechanisms of variable expressivity in
Neurofibromatosis Type 2-related Schwannomatosis. She completed her ABMGG
fellowship in Laboratory Genetics and Genomics at the National Human Genome Research
Institute and brings over a decade of combined clinical and research experience spanning
molecular pathology and genomic medicine.
Libby Williams
University of Plymouth
Libby Williams is a postdoctoral researcher at the University of Plymouth, UK, working as part of the Plymouth Brain Tumour Research Centre of Excellence. Her current research work focuses on the role of aldehyde dehydrogenase (ALDH) enzymes in NF2-null meningioma, funded by the Children’s Tumor Foundation Young Investigator Award program. This project follows on from her PhD research in the same topic area under the supervision of Prof. David Parkinson.
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Session Co-Chairs:
David Parkinson, PhD, University of Plymouth
Daochun Sun, PhD, Medical College of Wisconsin
3:00 PM - 3:25 PM: Invited Talk: From Early Formation to Precision Targeting: a glioma undertaking
Claudia Barros, PhD, University of Plymouth
3:25 PM - 3:50 PM: Invited Talk: From Stemness to Therapy: Leveraging Tumor Heterogeneity to Target pNF at Early Stages
Sajjad Khan, PhD, Medical College of Wisconsin
3:50 PM - 4:00 PM: Break
4:00 PM - 4:15 PM: Platform Talk: Auditory Phenotype in Neurofibromatosis Type 2–related Schwannomatosis is Associated with Schwann cell dysfunction and Peripheral Myelinopathy
Judith Kempfle, MD, UMass Chan Medical School/UMass Memorial Medical Center & Massachusetts Eye and Ear/Harvard Medical School
4:15 PM - 4:30 PM: Platform Talk: Sensory neuron activity promotes tumor progression in malignant peripheral nerve sheath tumor (MPNST) via TRPV1⁺ nociceptor signaling
Khalil Ali Ahmad Kasm, PhD, University of Texas MD Anderson Cancer Center
4:30 PM - 4:45 PM: Platform Talk: Exploring the role of the human microenvironment in Neurofibromatosis-related tumor formation and progression
Jonas Van Lent, PhD, Institute of Oncology Research (IOR)
4:45 PM - 5:00 PM: Platform Talk: Analysing the role and therapeutic potential of ALDH1A3 in NF2-null meningioma
Libby Williams, PhD, University of Plymouth
27 June, 2026 03:50 pm
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04:50 pm
West Coast RASopathy Clinician Affinity Group Meeting (Optional Satellite Meeting)
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A one-hour informal affinity group for west coast clinicians. The session will be facilitated by Dr. Tamar Green and will include light snacks and an open discussion on topics relevant to west coast clinicians caring for patients with RASopathies. Refreshments will be served
27 June, 2026 05:00 pm
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06:30 pm
Welcome Cocktail Reception
Centennial Foyer
A cocktail reception hosted by the Children's Tumor Foundation for all NF Conference attendees.
28 June, 2026 07:00 am
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03:00 pm
Registration & Check In
Centennial Foyer
Information & Help Desk is also open at this time.
28 June, 2026 07:00 am
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08:30 am
Breakfast
Centennial Foyer
Light breakfast is available to all registered attendees.
28 June, 2026 07:00 am
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08:30 am
Symposium: Bridging the Gap: Expert and Patient Perspectives on Transition and Continuity of Care for Those Living With NF1-PN
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Hosted by SpringWorks Therapeutics.
28 June, 2026 07:00 am
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08:20 am
Understanding Sleep in Neurofibromatosis Type 1: Integrating Clinical, Genetic, and Basic Science Research (Satellite Session)
James Walker
Massachusetts General Hospital
James Walker, PhD is an Assistant Professor of Neurology in the Center for Genomic Medicine at Massachusetts General Hospital and Harvard Medical School, and a member of the Broad Institute of MIT and Harvard. His laboratory studies the molecular and cellular mechanisms of neurofibromatosis type 1 (NF1) with the goal of identifying new therapeutic targets. His research integrates functional genomics and proteomics across Drosophila and human cell-based models to gain insight into disease mechanisms. In parallel, his lab investigates gene editing strategies for NF1 and examines sleep dysfunction as a key quality-of-life issue in affected individuals. Dr. Walker received his PhD from the University of Cambridge and completed postdoctoral training at Massachusetts General Hospital.
Alex Dyson
Massachusetts General Hopital
Alex Dyson currently works a postdoctoral research fellow in the Walker Lab at Massachusetts General Hospital, Boston, where he uses a Drosophila (fruit fly) model of NF1 to investigate the molecular mechanisms through which neurofibromin regulates neuronal function and behavior. This follows on from his graduate work with Richard Baines and Shruti Garg at the University of Manchester (UK), where he again used Drosophila to study the role of the NF1 gene in synaptic transmission.
Jadwiga Bilchak
University of Pennsylvania
Jadwiga's work focuses on the mechanisms of neurobehavioral symptoms in Neurofibromatosis Type 1, specifically in relation to sensory function. Using high-throughput behavior assays, in-vivo Ca2+ imaging, and the genetic manipulations available in the Drosophila model, she investigates how sensory abnormalities are linked to social behavior and sleep
Natalie Pride
University of Sydney
Dr. Natalie Pride is a clinical neuropsychologist who received her undergraduate and master’s degree at Macquarie University (Sydney, Australia). She completed her doctoral education at the University of Sydney and pursued postdoctoral training under Professor Kathryn North and A/Professor Jonathan Payne at the Children’s Hospital at Westmead (CHW), Australia. During this training her research focused on the neural correlates of cognitive, social and behavioral impairment in individuals with NF1. Currently, Dr. Pride divides her time between leading the NF1 Learning Clinic, where she specializes in the neuropsychological management of children with NF1 and heading the NF1 Neuropsychology Research Team at the Kids Neuroscience Centre, CHW. Dr. Pride was awarded a new investigator award from CDMRP NFRP to study sleep disturbances in children with NF1, risk factors and adverse cognitive outcomes. She is a Francis. S. Collins Scholar with her current research focusing on characterising sleep disorders using polysomnography and exploring causes and their impacts in NF1. With more than 17 years of clinical and translational research experience in NF1, Dr Pride has dedicated her career to understanding and improving diagnosis and treatment pathways for children and adolescents with NF1 with neuropsychological challenges.
Elizabeth Brown
Florida State University
Dr. Liz Brown is an Assistant Professor in the Department of Biological Science and Program in Neuroscience at Florida State University. Prior to joining FSU, Dr. Brown completed her PhD in Biological Science at the University of Cincinnati and a postdoctoral fellowship at Texas A&M University. Dr. Brown’s investigates the genetic and neural regulation of sleep and feeding behaviors using Drosophila as a model system. She is particularly interested in how the genes and neural circuits that regulate these behaviors are modified by context, including aging and disease. Her lab uses multiple approaches, including genetic screening, behavioral analysis, functional genomics, and microscopy. In addition to research, Dr. Brown is deeply committed to academic mentorship and STEM education.
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Session Chairs:
Natalie Pride, PhD, University of Sydney
James Walker, PhD, Massachusetts General Hospital
7:00 AM - 7:10 AM: Platform Talk: Insights into the role of NF1 in sleep and circadian activity from animal models and human genetics
Alex Dyson, PhD, Center for Genomic Medicine, Massachusetts General Hospital and Harvard Medical School
7:10 AM - 7:20 AM: Platform Talk: Neurofibromin 1 mediates sleep depth and metabolism in Drosophila
Elizabeth Brown, PhD, Florida State University
7:20 AM - 7:30 AM: Platform Talk: Insomnia, sleep health and sleep architecture in paediatric NF1: Clinical impact and biopsychosocial risk factors
Natalie A. Pride, PhD, University of Sydney
7:30 AM - 7:40 AM: Platform Talk: Longitudinal, objective measurement and analysis of sleep-wake patterns in patients with NF1
James A. Walker, PhD, Massachusetts General Hospital
7:40 AM - 7:50 AM: Platform Talk: Understanding Daily Sleep-Emotion Links in Adolescents with Neurofibromatosis Type 1
Dan Liu, PhD, Florida State University
7:50 AM - 8:00 AM: Platform Talk: Sensory integration and sleep in a Drosophila model of NF1
Jadwiga Bilchak, PhD, School of Medicine, University of Pennsylvania
8:00 AM - 8:20 AM: Q & A Panel & Discussion
Panelists:
Alex Dyson, PhD
Elizabeth Brown, PhD
Natalie A. Pride, PhD
James A. Walker Ph.D.
Dan Liu, PhD
Jadwiga N. Bilchak, PhD
Session Chairs:
Natalie Pride, PhD, University of Sydney
James Walker, PhD, Massachusetts General Hospital
28 June, 2026 08:45 am
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09:00 am
Award Presentation
Annette Bakker
Children's Tumor Foundation
Annette Bakker, a Ph.D. in Biochemistry, was an academic researcher for ten years - University Antwerp, Yale Medical School, and the Myology Institute Paris. Following this, she accumulated 15 years of experience in multiple executive leadership positions in Oncology R&D in big pharma and biotech. She holds over 50 publications and 5 patents (https://orcid.org/0000-0001-8420-7831).
Motivated by the realization that numerous groundbreaking discoveries fail to translate into clinical benefit, she joined the Children’s Tumor Foundation (CTF) in 2011 to deploy CTF's talent, time, and treasure (TTT) to help bridge the gaps between scientific discoveries and clinical benefits, particularly focusing on neurofibromatosis, a rare genetic disorder. In 2022, she was decorated Officer in the Order of Leopold by the king of Belgium for her bold approach and dedication to improving patients' lives.
Annette strongly believes that patient-centric research foundations hold a unique position in the R&D ecosystem. Organizations, such as CTF, are trusted partners for all stakeholders, with the same sense of urgency as the patients. As a FasterCures Changemaker and Chan Zuckerberg Initiative mentor, Annette is deeply committed to constructing an enterprise that not only benefits patients with NF but serves as a model for expediting drug discovery and development within the broader rare disease community.
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2026 Friedrich von Recklinghausen Award:
Presented by Annette Bakker, PhD, Children’s Tumor Foundation
28 June, 2026 09:00 am
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12:00 pm
Developmental Origins and Cellular Drivers of NF Disease, Session 2
Daochun Sun
Medical College of Wisconsin
Dr. Sun obtained his PhD degree in Molecular Biology and Genetics by studying mechanisms of Neurofibromatosis Type 1 (NF1)-associated Malignant Peripheral Nerve Sheath Tumors (MPNST) at School of Medicine, Wayne State University. He further received postdoctoral training from the Developmental Biology Department at the University of Texas, Southwestern Medical Center, and Cancer Biology and Genetics program in Memorial Sloan Kettering Cancer Center. His works emphasize the cell-of-origins of the tumor, and he identified a stem-like cell population playing essential roles in tumorigenesis, relapse, and metastasis of NF1-associated plexiform neurofibromas and MPNST. These discoveries may provide novel strategies to prevent tumor transformation, progression, chemoresistance, and metastasis.
Eduard Serra-Arenas
Germans Trias i Pujol Research Institute (IGTP)
Dr. Eduard Serra leads the Hereditary Cancer Group at the Germans Trias i Pujol Research Institute (IGTP), in Badalona (Barcelona), Spain. His group is part of the Cancer Translational Research Program (CARE) at IGTP. Research in his laboratory focuses on the study of tumors associated with Neurofibromatosis Type 1 (NF1), particularly those that arise in the peripheral nervous system. The group studies their development, composition, progression and possible therapies and patient management. The levels of study are the genomic analysis of tumors and the generation and the in vitro and in vivo use of cellular models, including 3D models derived from induced pluripotent stem cells (iPSCs). He is a close collaborator of different local and international associations of patients with NF1.
Daniel Snellings
Boston Children's Hospital
Daniel Snellings is a postdoctoral fellow with Christopher Walsh at Boston Children’s Hospital. He completed his PhD with Douglas Marchuk at Duke university studying how somatic mutations drive vascular malformations. His research at Boston Children’s is focused on understanding the role of somatic mutations in NF1 pathogenesis.
Manuel Bettencourt
Mayo Clinic
Manuel Bettencourt is a Post-Baccalaureate Research Scholar in the Post-Baccalaureate Research Education Program (PREP) at Mayo Clinic, where he works in the laboratories of Dr. Sheila Mansouri and Dr. Gelareh Zadeh. PREP is a mentored post-baccalaureate research training program for recent graduates preparing for advanced biomedical, medical, or physician-scientist training.
Manuel’s research focuses on computational and translational precision oncology, integrating bulk transcriptomic, single-cell, spatial, molecular, and clinical data to study cancer biology and treatment response. His current work investigates neurofibromatosis, cutaneous neurofibromas, peripheral nerve sheath tumors, and glioblastoma, with emphasis on tumor microenvironment remodeling, Schwann-cell developmental states, and clinical trial emulation.
He earned his B.S. in Quantitative Science from Emory University with Highest Honors. His prior work includes single-cell cancer biomarker discovery and development of CAMP, a computational framework for single-cell lineage tracing and metastatic profiling. His long-term goal is to build multi-modal tools that improve individualized cancer risk prediction and therapeutic decision-making.
Centennial DE
Session Co-Chairs:
David Parkinson, PhD, University of Plymouth
Daochun Sun, PhD, Medical College of Wisconsin
9:00 AM - 9:25 AM: Invited Talk: Developmental Vulnerability and Therapeutic Opportunities for NF1-associated Tumors in the Nervous System
Yuan Zhu, PhD, UT Southwestern Medical Center
9:25 AM - 9:50 AM: Invited Talk: iPSC-based merlin-deficient Schwann cell-like spheroids as a new in vitro model system for studying the pathogenesis of NF2-SWN Schwann cells and for evaluating new therapeutic approaches
Elisabeth Castellanos, PhD, German Trias i Pujol Research Institute (IGTP)
9:50 AM - 10:05 AM: Platform Talk: Pervasive Somatic Loss of NF1 in Non-Tumor Brain Tissue Drives Glial Expansion in Neurofibromatosis Type 1
Daniel Snellings, PhD, Boston Children’s Hospital
10:05 AM - 10:20 AM: Platform Talk: A CRISPR-based Zebrafish Model of NF1 that Provides Novel Insights into CNS Tumors
Benjamin Callaway, PhD, Miami University
10:20 AM - 10:30 AM: Break
10:30 AM - 10:45 AM: Platform Talk: iPSC-derived NF1-CDKN2A-PRC2 deficient neural crest mimics
Eduard Serra, PhD, Germans Trias i Pujol Research Institute (IGTP)
10:45 AM - 11:00 AM: Platform Talk: Germline NF2 variant position constrains somatic second hits and determines clinical severity in Neurofibromatosis Type 2-related Schwannomatosis
Niveditha Ravindra, MD, National Institutes of Health
11:00 AM - 11:15 AM: Platform Talk: Functional merlin depletion defines a TEAD1-dependent Schwann cell state driving schwannoma progression
Maxwell Laws, MD, National Institutes of Health
11:15 AM - 11:30 AM: Platform Talk: Developmental Heterogeneity of Schwann Cells in Cutaneous Neurofibromas Revealed by Bulk and Single-Nucleus RNA Sequencing
Manuel Bettencourt, BSc, Mayo Clinic
11:30 AM - 11:45 AM: Platform Talk: Epigenetic Subgroups and a Schwann Dedifferentiation Axis Define Clinical and Transcriptional Heterogeneity in MPNST
Chloe Gui, MD, University of Toronto
28 June, 2026 12:00 pm
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01:00 pm
Lunch
Centennial Foyer
Box lunches are available for purchase daily, Saturday through Tuesday. To save time and avoid on-site concession lines, please purchase your box lunch(es) during registration.
28 June, 2026 12:15 pm
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01:00 pm
Transitioning Pediatric Patients with Neurofibromatosis Type 1 (NF1) and Plexiform Neurofibromas (PN) to Adult Care
Centennial GH
Lauren Weintraub, MD, Albany Medical Center
Recognize and act early on pediatric NF1 plexiform neurofibromas. We will use concise case vignettes to illustrate decision points for imaging, surgery or pharmacological treatment, and supportive care, while outlining patient education, insurance navigation, copay assistance, and community connections. The session will also clarify body surface area (BSA)-based dosing, drug administration, and drug interactions.
28 June, 2026 12:15 pm
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12:45 pm
NF AI Innovation Showcase (Satellite)
Centennial F
Join us in Centennial F during lunch for a series of rapid-fire pitches and live demos. Each team will have 3–5 minutes to present proof-of-concept projects they built in Saturday’s workshop, highlighting core scientific questions and upcoming milestones. Coaches, expert guests, and peers will provide immediate feedback.
28 June, 2026 01:00 pm
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02:00 pm
Keynote: How Efforts to Understand Metabolism Inform Neoplasia Progression
Matthew Vander Heiden
Massachusetts Institute of Technology
Matthew Vander Heiden is the Director of the Koch Institute for Integrative Cancer Research, the Lester Wolfe Chair in Molecular Biology, and a Professor in the Department of Biology at the Massachusetts Institute of Technology. He is also a practicing Medical Oncologist and Instructor of Medicine at the Dana-Farber Cancer Institute and Harvard Medical School, as well as an Institute Member of the Broad Institute of Harvard and MIT. Vander Heiden received his MD and PhD degrees from the University of Chicago, where he worked in the laboratory of Craig Thompson. He completed clinical training in internal medicine at the Brigham and Women’s Hospital and a Hematology-Oncology fellowship at the Dana-Farber Cancer Institute / Massachusetts General Hospital program. He was a post-doctoral fellow in the laboratory of Lewis Cantley at Harvard Medical School. In 2010, Vander Heiden joined the MIT faculty. His laboratory studies how metabolism is regulated to meet the needs of cells in different physiological situations. A major focus of his research is the role of metabolism in cancer. Using a combination of biochemistry, molecular biology and mouse models, the aim of the Vander Heiden laboratory is to understand how metabolism influences different stages of tumor biology with a goal to improve cancer treatment and enable the earlier detection of cancer. He also serves on the scientific advisory boards of multiple cancer centers, as well as the investment advisory board and scientific advisory boards of multiple companies.
Centennial DE
Matthew Vander Heiden, MD, PhD, Massachusetts Institute of Technology
28 June, 2026 02:00 pm
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05:00 pm
Altered Metabolism in NF
Alexandra O'Donohue
University of Sydney
Dr Alexandra O’Donohue is an early career postgraduate researcher from the Bioengineering & Molecular Medicine laboratory at the Westmead Institute for Medical Research and the University of Sydney. Dr O'Donohue received her BMedSci(Hons) in molecular biology and genetics in 2018 and then went on to complete her PhD from the University of Sydney in 2023. Throughout her postgraduate studies, she cultivated a keen interest in gene therapy, developing an expertise in CRISPR editing and the design, generation, and screening of tissue-specific recombinant adeno- associated vectors. Through mentorship with A/Prof Aaron Schindeler, she has cultivated her research interested in Neurofibromatosis type 1 and NF2-related Schwannomatosis. She has continued a body of work examining the links between metabolic changes associated with NF1 muscle weakness in bone, as well as expanded her gene therapy efforts for NF2-related Schwannomatosis. In 2025 she was awarded a fellowship via the US Children’s Tumor Foundation Young Investigator Award to support her research program.
James Walker
Massachusetts General Hospital
James Walker, PhD is an Assistant Professor of Neurology in the Center for Genomic Medicine at Massachusetts General Hospital and Harvard Medical School, and a member of the Broad Institute of MIT and Harvard. His laboratory studies the molecular and cellular mechanisms of neurofibromatosis type 1 (NF1) with the goal of identifying new therapeutic targets. His research integrates functional genomics and proteomics across Drosophila and human cell-based models to gain insight into disease mechanisms. In parallel, his lab investigates gene editing strategies for NF1 and examines sleep dysfunction as a key quality-of-life issue in affected individuals. Dr. Walker received his PhD from the University of Cambridge and completed postdoctoral training at Massachusetts General Hospital.
Andrea Santangelo
University of Genoa
Andrea Santangelo, MD, PhD(c) is a pediatrician and physician-scientist with a clinical and research focus on
pediatric neurology, neurocutaneous disorders, and translational neuroscience. He is currently an attending
physician at the Pediatric Department of the Children’s Hospital “G. Di Cristina” in Palermo, Italy, and a PhD
candidate in the IRCCS Istituto Giannina Gaslini, in collaboration with University of Genoa.
His research is primarily dedicated to neurocutaneous syndromes, mainly, Neurofibromatosis type 1, with a focus
on identifying prognostic biomarkers and improving treatment monitoring in patients with plexiform neurofibromas.
He is Principal Investigator of clinical and translational studies exploring new approaches and real-world
effectiveness of targeted therapies.
Dr. Santangelo has authored several peer-reviewed publications, spanning pediatric neurology, neuro-oncology,
epilepsy, and metabolic and inflammatory pathways in neurological diseases. His work integrates clinical data with
pharmacokinetics, advanced imaging, and omics-based approaches to better characterize variability in tumor
behavior and therapeutic outcomes.
A major focus of his work is MEK inhibition in NF1, with an emphasis on the clinical and biological determinants
of treatment response. His research explores interindividual variability in selumetinib pharmacokinetics and
metabolism, contributing to the development of therapeutic drug monitoring strategies and more personalized
treatment approaches.
In parallel, he investigates systemic metabolic alterations in NF1, demonstrating that MEK inhibition is associated
with a distinct metabolic reprogramming involving energy metabolism and mitochondrial pathways, with potential
implications for biomarker discovery and treatment monitoring.
Dr. Santangelo’s work lies at the intersection of clinical care and translational innovation, with particular interest in
integrating imaging biomarkers, pharmacokinetics, and systems biology to better understand disease progression
and therapeutic response in neurocutaneous syndromes and related conditions. His current research aims to bridge
preclinical insights and real-world clinical data to advance precision medicine approaches in pediatric neuro-
oncology.
Bavani Subramaniam
Children's National Hospital
I am a postdoctoral research fellow at the Center for Cancer and Immunology Research at Children’s National Hospital in Washington, DC. I specialize in molecular oncology and pharmaceutical technology, with expertise in preclinical cancer models, targeted therapies, and drug delivery systems. My current research focuses on uncovering mechanisms of resistance and vulnerabilities in pediatric high-grade gliomas, including NF1- and MTAP-deficient tumors, and evaluating novel therapeutic strategies. I have authored multiple peer-reviewed publications in high-impact journals and am a recipient of several competitive awards, including the Lilabean Fellowship and the Children’s Tumor Foundation Drug Discovery Initiative.
Charlotte Ohl
Fritz Lipmann Institute
My name is Charlotte Ohl and I am a doctoral student at the research group of Prof. Dr. Helen
Morrison at the Fritz Lipmann Institute – Leibniz Institute on Aging in Germany. I received my
bachelors degree in biology with focus on molecular biology at the Friedrich-Schiller-University
Jena, followed by my masters degree in molecular medicine. I am interested in LZTR1-related
pathologies and currently working on understanding how peripheral nerve pathologies associated
with LZTR1 affect inter-organ communication and metabolic homeostasis. My research is
supported by a fellowship from the Leibniz Graduate School of Aging (LGSA).
Kathryn Lemberg
Johns Hopkins University
Dr. Kathryn Lemberg is a pediatric oncologist and physician-scientist in the Sidney Kimmel Comprehensive Cancer Center at the Johns Hopkins University School of Medicine. Dr. Lemberg’s research investigates metabolism in childhood cancer, with a focus on improving treatments and outcomes for patients with pediatric sarcomas and cancer predisposition syndromes, including neurofibromatosis type I (NF1). She leads a translational research lab investigating the interactions between tumor metabolism and oncogenic signaling pathways in models of NF1-deficient and RAS-active cancers, including malignant peripheral nerve sheath tumor, rhabdomyosarcoma, and glioma. Dr. Lemberg’s group uses small molecule metabolite antagonists to perturb tumor metabolism and study the consequences for tumor biology in cell culture and xenograft models. In addition, Dr. Lemberg investigates the interactions between systemic metabolism and tumor growth in animal models and retrospective cohorts of patients with NF1, with the goal of optimizing supportive management for this population of patients. She collaborates with clinicians and researchers at Johns Hopkins and elsewhere to carry out these studies. Dr. Lemberg received her undergraduate degree from the University of Chicago and completed her graduate and medical training in the Medical Scientist Training Program at Columbia University, College of Physicians and Surgeons. She completed a residency in general pediatrics at Johns Hopkins, and then went on to complete fellowship training in pediatric hematology/oncology in the joint Johns Hopkins-National Cancer Institute training program. She was a member of the Johns Hopkins Drug Discovery group for her postdoctoral fellowship training. Dr. Lemberg’s research has been generously supported by funding from several sources including a CureSearch for Children’s Cancer Young Investigator Award, a Hyundai Hope on Wheels Young Investigator Award, a Cannonball Kids’ Cancer Foundation Young Investigator Grant, a Tap Out Cancer Award, and a Neurofibromatosis New Investigator Award from the Congressionally Directed Medical Research Program. In 2025 she received the Director’s Teaching Award for Pediatric Oncology at Johns Hopkins and was a selected fellow for the NCI-funded Transdisciplinary Research on Energetics and Cancer training workshop.
Matthew Vander Heiden
Massachusetts Institute of Technology
Matthew Vander Heiden is the Director of the Koch Institute for Integrative Cancer Research, the Lester Wolfe Chair in Molecular Biology, and a Professor in the Department of Biology at the Massachusetts Institute of Technology. He is also a practicing Medical Oncologist and Instructor of Medicine at the Dana-Farber Cancer Institute and Harvard Medical School, as well as an Institute Member of the Broad Institute of Harvard and MIT. Vander Heiden received his MD and PhD degrees from the University of Chicago, where he worked in the laboratory of Craig Thompson. He completed clinical training in internal medicine at the Brigham and Women’s Hospital and a Hematology-Oncology fellowship at the Dana-Farber Cancer Institute / Massachusetts General Hospital program. He was a post-doctoral fellow in the laboratory of Lewis Cantley at Harvard Medical School. In 2010, Vander Heiden joined the MIT faculty. His laboratory studies how metabolism is regulated to meet the needs of cells in different physiological situations. A major focus of his research is the role of metabolism in cancer. Using a combination of biochemistry, molecular biology and mouse models, the aim of the Vander Heiden laboratory is to understand how metabolism influences different stages of tumor biology with a goal to improve cancer treatment and enable the earlier detection of cancer. He also serves on the scientific advisory boards of multiple cancer centers, as well as the investment advisory board and scientific advisory boards of multiple companies.
Pavlina Sverak
Department of Pediatric Hematology/Oncology, the Largaespada Lab, and the Brain Tumor Program at the University of Minnesota
Dr. Sverak received her MD from Charles University in Prague, Czech Republic. She is a researcher affiliated with the Department of Pediatric Hematology/Oncology, the Largaespada Lab, and the Brain Tumor Program at the University of Minnesota. Her research focuses on Neurofibromatosis type 1 (NF1), with particular interest in the role of inflammation in NF1 tumorigenesis. Her work aims to identify low-toxicity dietary and nutraceutical interventions that may regulate inflammatory status in individuals with NF1 and potentially alter disease progression.
Miriam Bornhorst MD
Lurie Children's Hospital of Chicago
Miriam Bornhorst has a joint appointment at George Washington University Hospital and Children’s National Hospital (CNH) in Washington D.C. Dr. Bornhorst’s main clinical interests are cancer predisposition (including Neurofibromatosis) and Neuro-Oncology. She is the Medical Director of the Cancer Genetics Clinic and Clinical director of the Gilbert Neurofibromatosis Program at CNH. Dr. Bornhorst is the institution PI for multiple clinical trials for patients with NF1 at CNH, including clinical trials of MEK-inhibitors. Her research focuses on early detection and treatment of patients at risk for developing cancer - ultimately improving long term outcomes. She is currently studying how MEK-inhibitor treatment affects metabolism in children with NF1, with the goal to identify biomarkers that can be used to predict response to treatment, as well as gain a better understanding of how diet affects tumor growth. She is also investigating novel and low allele fraction genomic changes within NF-associated tumors using optical genome mapping.
Stephanie Jaramillo
Stephanie Jaramillo, MS, is a Colorado native, licensed clinician, advocate, wife, and mother of three. She earned her Master of Science in Marriage and Family Therapy from the University of Phoenix and has been serving individuals, couples, and families throughout Colorado since 2013.
Stephanie's professional and personal experiences have shaped her passion for supporting families navigating trauma, chronic illness, disability, life transitions, addiction, relationship challenges, and the complexities of caregiving. As the mother of twins and a son living with Neurofibromatosis (NF), she understands firsthand the emotional, medical, and systemic challenges families face when caring for a loved one with a rare disease or disability.
Beyond her clinical work, Stephanie is a nationally recognized advocate and volunteer leader with the Children's Tumor Foundation. Through her advocacy efforts at both the state and national levels, she has worked to advance awareness, research, and access to care for individuals living with rare diseases and disabilities. Her dedication to service has helped raise significant funds for research, support countless families, and amplify the voices of those often underserved within healthcare systems.
Stephanie brings authenticity, compassion, and lived experience to every aspect of her work, combining professional expertise with a deep commitment to empowering individuals, strengthening families, and creating meaningful change within her community.
Semira Ortiz
Pennington Biomedical Research Center
Centennial DE
Session Co-Chairs:
Miriam Bornhorst, MD, Lurie Children's Hospital
Kathryn Lemberg, MD, PhD, Johns Hopkins University
Part I: Tumor metabolism in NF
2:00 PM - 2:15 PM: Caregiver Perspective
Stephanie Jaramillo, NF1 Parent & Advocate
2:15 PM - 2:30 PM: Invited Talk: Metabolic Reprogramming in Neurofibromatosis Type 1: Plasma Metabolomic Signatures of MEK Inhibition
Andrea Santangelo, MD, University of Genoa
2:30 PM - 2:45 PM: Invited Talk: Targeting glutamine-dependent pyrimidine synthesis as a therapeutic approach for NF1 and RAS-mutant sarcomas
Kathryn Lemberg, MD, PhD, Johns Hopkins University
2:45 PM - 3:00 PM: Platform Talk: Phase I Study of a Nutraceutical Intervention with High-Phenolic Extra-Virgin Olive Oil and Curcumin in Neurofibromatosis Type 1
Pavlina Sverak, MD, University of Minnesota
3:00 PM - 3:15 PM: Platform Talk: Targeting PRMT5 in MTAP-Deficient NF1 High-Grade Gliomas Suppresses DNA Repair Pathways and Enhances Sensitivity to MEK Inhibition
Bavani Subramaniam, Children’s National Hospital
3:15 PM - 3:30 PM: Platform Talk: Superoxide dismutase: a novel therapeutic target for treating MPNST
Somaiah Chinnapaka, PhD, University of Virginia
3:30 PM - 3:45 PM: Break
Part II: Systemic metabolism in NF
3:45 PM - 4:00 PM: Invited Speaker: Dietary intervention ameliorates the high bone porosity seen in a murine model of NF1
Alexandra O'Donohue, PhD, University of Sydney
4:00 PM - 4:15 PM: Platform Talk: Evaluating metabolic alterations in an NF1 mouse model
Alyssa Hohman, PhD, Northwestern University
4:15 PM - 4:30 PM: Platform Talk: The role of LZTR1 at the nerve-organ interface and its effects on metabolic regulation
Charlotte Ohl, MSc, Fritz Lipmann Institute
4:30 PM - 4:45 PM: Platform Talk: Neurofibromin Regulates Systemic Growth and Metabolic Homeostasis via Neuronal Control of Carbohydrate and Lipid Metabolic Programs
James A. Walker, PhD, Massachusetts General Hospital
4:45 PM - 5:00 PM: Platform Talk: NF1 loss of heterozygosity causes impaired mitochondrial respiration in a mouse model of a patient mutation
Semira Ortiz, PhD, Pennington Biomedical Research Center
28 June, 2026 02:00 pm
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05:00 pm
Deconstructing Complexities & Burdens of NF-Related Pain (Concurrent Session)
David Largaespada
University of Minnesota
Dr. David A. Largaespada is Professor in the Departments of Pediatrics and Genetics, Cell Biology & Development and the Deputy Director of the Masonic Cancer Center at the University of Minnesota, where he also co-directs the Brain Tumor Program and the Center for Genome Engineering. He holds the Hedberg Family/Children’s Cancer Research Fund Chair in Brain Tumor Research. Dr. Largaespada is a leading authority in cancer genetics, functional genomics, and translational modeling, with more than 30 years of continuous research on Neurofibromatosis Type 1 (NF1)–associated tumors. His laboratory has shown that GM-CSF and STAT5 signaling drive juvenile myelomonocytic leukemia (JMML) in NF1, that WNT and SHH signaling define two distinct subtypes of malignant peripheral nerve sheath tumor (MPNST), and that PTEN loss and PI3K activation promote MPNST progression. He has developed novel mouse and human iPSC-based models of plexiform neurofibroma, atypical neurofibroma (ANNUBP), and MPNST, and applied Sleeping Beauty transposon mutagenesis to elucidate the stepwise genetic evolution of these tumors. His group also employs forward genetic and pharmacologic screening to identify synthetic lethal targets and strategies to enhance adoptive cell therapies for cancer. A recipient of the American Cancer Society Research Professor Award, Dr. Largaespada has co-founded four biotechnology companies translating genetic discoveries into clinical innovation.
Larry Sherman
Oregon Health and Sciences University
Dr. Larry S. Sherman is the Assistant Chief and a Professor in the Division of Neuroscience at the Oregon National Primate Research Center and in the Neuroscience Graduate Program at the Oregon Health & Science University (OHSU). He is also the President of the Oregon and Southwest Washington Chapter of the Society for Neuroscience. He has over 120 publications related to brain development and neurodegenerative diseases including studies on neurofibromatosis and schwannomatosis. The Oregon Museum of Science and Industry and Portland Monthly Magazine recognized Dr. Sherman as one of the most innovative people in the State of Oregon. In 2012, he was recognized by the OHSU School of Medicine Faculty Senate and the OHSU foundation for Outstanding Teaching, and was awarded the Mary Omberg Award for outstanding support of science education in Oregon and Southwest Washington.
Steven Rhodes
Dr. Steven Rhodes is an Assistant Professor of Pediatrics at the Indiana University School of Medicine where he directs the Neurofibromatosis (NF) Multidisciplinary Program at the IU Simon Comprehensive Cancer Center. He earned his MD and PhD from the Medical Scientist Training Program at Indiana University, followed by residency training in Pediatrics and fellowship training in Pediatric-Hematology Oncology at Riley Hospital for Children, where he conducted his postdoctoral research in the laboratory of Wade Clapp, MD. Dr. Rhodes has a scientific focus on the development of genetically engineered mouse models that recapitulate the progression of atypical neurofibromas and transformation to malignant peripheral nerve sheath tumor (MPNST) in NF1. Using these models and state-of-the-art approaches in single cell analytics, Dr. Rhodes is working to identify potential therapeutic approaches to both treat and ultimately even prevent MPNST development. Dr. Rhodes’s research is supported by the Francis S. Collins Scholars Program in Neurofibromatosis Clinical and Translational Research funded by the Neurofibromatosis Therapeutic Acceleration Program (NTAP).
Dale Berg
Patient Advocate
Dale Berg is a patient representative with REiNS(Response Evaluation in Neurofibromatosis and Schwannomatosis), active with Children’s Tumor Foundation local NF Symposiums, Clinical Care Advisory Board, CTF NF Walk, NF Summit and Conferences. He has also participated in Cupid’s Undie Run. He was diagnosed in 2015 with Schwannomatosis at age 45. His journey began in 2005 when he had an MRI due to increasing lower back pain. Two consultations by spinal surgeons diagnosed the pain due to a degenerative disc. He ignored increasing pain and discomfort for another 10 years. During a routine doctor visit, his general practitioner urged for updated imaging. Multiple tumors were found and the consulting surgeon had a vague knowledge that the multiple schwannomas that were found could be related to NF. After a quick internet search for NF, Dale was fortunate to have the University of Minnesota and Mayo Rochester NF clinics. Schwannomatosis was confirmed at Mayo. Dr. Dusica Babovic-Vuksanovic introduced him to patient advocacy with a referral to REiNS in 2017. Dale and his wife have a 14-year old son and 17 year old daughter and live in Minnesota.
David Pang
Guys and St Thomas' Hospital NHS Trust
I have been a Consultant in Pain Management and anaesthesia at St Thomas’ Hospital since 2012. My initial journey in medicine was in paediatrics and then I started anaesthesia training in 2004. During this time, I developed an interest in pain medicine and managing patients with chronic persistent pain in both adults and children. I am part of the national multidisciplinary neurofibromatosis team at Guy’s Hospital London UK and I help manage patients with neurofibromatosis and schwannomatosis related chronic pain. My research interest is in developing multidisciplinary approaches to manage chronic pain in adults and children and neurostimulation for refractory neuropathic pain.
Deepak Ravindran
Teesside University, School of Health & Life Sciences
Deepak is an Honorary Professor at Teesside University, UK, and a Consultant in Pain and Lifestyle Medicine. He is the Founder and Director of the Berkshire Pain Clinic and previously served as Clinical Lead for Pain Medicine at the Royal Berkshire NHS Foundation Trust. He is the author of the Amazon bestselling book The Pain-Free Mindset, which bridges modern pain science with practical, patient-centred recovery strategies.
Deepak is recognised for his trauma-informed, “upstreamist” approach to pain care—shifting the focus from symptom control to addressing root causes across biological, psychological, and social domains. He has led the development of award-winning NHS services for Pain and Longcovid in Berkshire county, UK, both of which have been recognised for innovative, whole-person care delivery at scale.
He serves on the council of the British Pain Society and is a trustee of the British Society of Lifestyle Medicine. His work spans clinical practice, education, service design, and digital health innovation. He is also a widely followed clinician-educator, with his content recognised by the World Health Organization and the Academy of Medical Royal Colleges as a trusted source of health information.
In his clinical and academic work, Deepak integrates advances in pain neuroscience, predictive processing models, and lifestyle medicine to develop scalable approaches for chronic pain and complex conditions. His MINDSET framework—encompassing medications, interventions, neuroscience education, diet, sleep, exercise, and mind-body therapies—underpins his approach to comprehensive pain care.
His talk at the conference will explore how integrating trauma-informed principles with lifestyle medicine can transform outcomes for individuals living with persistent pain, including those with complex neurogenetic conditions.
Heather Thompson
California State University, Sacramento
Heather L. Thompson, PhD., CCC-SLP (she/her) is a licensed Speech-Language Pathologist (SLP), Professor, and Department Chair of the Department of Communication Sciences and Disorders at California State University, Sacramento. Since 2015 she has taught, and since 2021, she has served as faculty coordinator for the Second Bachelor’s Degree in Communication Sciences and Disorders offered through the College of Continuing Education at Sacramento State.
She serves as the SLP Discipline Director for the Northern California Leadership Education in Neurodevelopmental Disabilities (NorCal LEND) program, which is housed at the University of California, Davis MIND Institute. She also holds a Language, Speech and Hearing Services Credential and provides per diem services as a speech-language pathologist at Sacramento City Unified School District and a Children’s Hospital in Northern California.
She has an interest in interdisciplinary clinical training for practitioners serving individuals with neurodevelopmental concerns. Her research interests include clinical trials and patient reported outcome measures. She has a specific interest in speech-language development of children with neurodevelopmental concerns including neurofibromatosis and the other RASopathies and evaluating assessments and interventions for and with individuals from these populations.
Kimberly Ostrow
Johns Hopkins School of Medicine
Dr. Kimberly Ostrow is a tumor biologist and neuroscientist whose research focuses on the mechanisms of pain
in schwannomatosis and related peripheral nerve tumors. She is an Assistant Professor in the Department of
Neurology at Johns Hopkins University and is recognized for her work on tumor-associated pain signaling.
Dr. Ostrow directs the Johns Hopkins Peripheral Nerve Sheath Tumor Tissue Bank, which collects tissue from
SMARCB1-related schwannomatosis, LZTR1-related schwannomatosis, NF2-related schwannomas, and
sporadic single schwannomas. Using these patient-derived samples, her laboratory developed the first human
schwannomatosis tumor cell lines and established one of the first in vivo models of schwannomatosis-
associated chronic pain.
Her research identified a key biological mechanism underlying tumor-associated pain: painful schwannoma
cells release cytokines and other signaling factors that sensitize nearby sensory neurons and promote
mechanical hypersensitivity. This work received recognition from NF Patients United at the 2020 European
Neurofibromatosis meeting for its potential clinical impact and was presented with a Public Day award.
Building on these discoveries, Dr. Ostrow is advancing translational approaches aimed at treating tumor-
associated pain. Her work has demonstrated that inhibition of mechanosensitive ion channels, with the peptide
GsMTx-4, which she helped characterize and clone during her postdoctoral training at SUNY Buffalo, can
reduce pain behaviors in preclinical models of schwannomatosis. These studies were supported through a
Children's Tumor Foundation Drug Discovery Initiative Award.
In addition to her neurofibromatosis research, Dr. Ostrow is actively involved in the broader pain research
community through participation in the United States Association for the Study of Pain and the International
Association for the Study of Pain. She has presented her findings at national and international scientific
meetings to increase awareness of pain mechanisms in neurofibromatosis and schwannomatosis.
Dr. Ostrow is also committed to mentorship, education, and patient-engaged research initiatives. Her work
continues to advance understanding of tumor-associated pain biology and support the development of new
therapies for patients living with schwannomatosis.
Namrata Raut
Cincinnati Children's Hospital Medical Center
Dr. Namrata G.R. Raut is a research fellow in the Jankowski lab at Cincinnati Children’s
Hospital Medical Center, investigating the role of glial cells in the onset of pain.
As a Young Investigator Award recipient from the Children’s Tumor Foundation, Dr. Raut
has focused on understanding the role of glial cell line-derived neurotrophic factor (GDNF)
released by Schwann cells and its contribution to the onset of pain in neurofibromatosis
type 1 (NF1).
In her current research, she aims to identify potential therapeutic targets for NF1 patients
experiencing pain to improve their daily quality of life.
Pamela Wolters
National Cancer Institute
Pam Wolters, PhD is a licensed psychologist and senior associate scientist in the Pediatric Oncology Branch of the National Cancer Institute, National Institutes of Health. She serves as the Director of the Health Psychology and Neurobehavioral Research Program and Co-Director of the Behavioral Health Core. Dr. Wolters leads a clinical research program that characterizes the longitudinal effects of disease and treatment on neuropsychological functioning and quality of life (QOL) in children and adults with chronic illness, including cancer and neurofibromatosis type 1 (NF1). One of her main areas of research is to identify, develop, and conduct appropriate clinical outcome assessments for natural history studies and treatment trials. For example, she is leading multi-center studies to develop and validate patient-reported outcome (PRO) measures that assess the chronic pain related to plexiform neurofibromas and to evaluate the use of an innovative device that objectively measures pain and sensory problems in children and adults with NF1. Dr. Wolters is the past chair and current member of the REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) PRO Working Group and member of the Leadership Council and Neurocognitive Working Group. She also is the Chair of the QOL Committee and member of the Steering and Neurocognitive Committees of the NF Clinical Trials Consortium, and Co-Chair of the Response Assessment in Pediatric Neuro-oncology PRO Committee.
Ignacio Blanco
Hospital Germans Trias i Pujol, Barcelona, Spain
Ignacio Blanco, M.D., Ph.D. practices clinical genetics at the Germans Trias Hospital. Dr. Blanco completed his residency in General and Digestive Surgery at Sant Pau Hospital (Barcelona, Spain), and completed a research fellowship in Medical Oncology and Immunotherapy at University of Nebraska Medical School, Omaha, Nebraska, USA. He is Spanish board-certified in General and Digestive Surgery and accredited in Human Genetics by the Spanish Society of Human Genetics.
Centennial GH
Session Co-Chairs:
Dale Berg, Patient Advocate
David Pang, MD, ChB, Guys and St Thomas' Hospital NHS Trust
2:00 PM - 2:05 PM: Welcome and Session Overview
Dale Berg, Patient Advocate
David Pang, MD, ChB, Guys and St Thomas' Hospital NHS Trust
2:05 PM - 2:25 PM: Invited Talk: Pain assessment in populations with varied communication needs
Heather Thompson, PhD, CCC-SLP, S-LP(C), California State University, Sacramento
2:25 PM - 2:40 PM: Platform Talk: Psychometric validation of novel electronic patient-reported outcome (PRO) measures to assess pain related to plexiform neurofibromas (pNF) in adults with neurofibromatosis type 1 (NF1)
Pamela Wolters, PhD, National Cancer Institute
2:40 PM - 2:55 PM: Platform Talk: Adult patients' experiences with selumetinib treatment versus placebo for neurofibromatosis type 1-plexiform neurofibroma (NF1-PN) associated symptoms and their impacts: a qualitative sub-study of a phase 3, placebo-controlled trial (KOMET)
Ignacio Blanco, MD, PhD, Hospital Germans Trias i Pujol, Barcelona, Spain
2:55 PM - 3:10 PM: Platform Talk: Mechanosensitive Ion Channel Inhibition Provides Durable, Tumor-Selective Analgesia in Schwannomatosis
Kimberly Ostrow, PhD, Johns Hopkins School of Medicine
3:10 PM - 3:25 PM: Break
3:25 PM - 3:50 PM: Invited Talk: Moving to Comprehensive Pain Care: A Trauma Informed Lifestyle Medicine Focused Approach
Deepak Ravindran, MD, Teesside University, School of Health & Life Sciences
3:50 PM - 4:05 PM: Platform Talk: GDNF Signaling as a Driver of Pain in Neurofibromatosis 1
Namrata Raut, PhD, Cincinnati Children's Hospital Medical Center
4:05 PM - 4:30 PM: Invited Talk: Distinct mechanisms of pain in neurofibromatosis and schwannomatosis
Larry Sherman, PhD, Oregon Health and Sciences University
4:30 PM - 4:55 PM: Panel Discussion & Q&A
Moderator:
David Pang, MD, ChB, Guys and St Thomas' Hospital NHS Trust
Panelists:
Heather Thompson, PhD, CCC-SLP, S-LP(C)
Pamela Wolters, PhD
Ignacio Blanco, MD, PhD
Kimberly Ostrow, PhD
Deepak Ravindran, MD; Namrata Raut, PhD
Larry Sherman, PhD
28 June, 2026 05:00 pm
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05:15 pm
Poster Advertisements
Centennial DE
Twelve Semi-Finalists for the 2026 NF Conference Poster Competition will present a one-slide, one-minute pitch (‘poster advertisement’) to Conference attendees.
28 June, 2026 05:30 pm
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08:00 pm
Poster Sessions & Cocktail Reception
Centennial Foyer
Basic / Preclinical Science and Clinical Science Poster Presentations. Refreshments will be served.
Tonight's poster session is split:
Session A: 5:30 PM – 6:45 PM
Presenters assigned to Session A should be available at their posters during this time.
Session B: 6:45 PM – 8:00 PM
Presenters assigned to Session B should be available at their posters during this time.
29 June, 2026 07:00 am
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01:00 pm
Registration & Check In
Centennial Foyer
Information & Help Desk is also open at this time.
29 June, 2026 07:00 am
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08:30 am
Breakfast
Centennial Foyer
Light breakfast is available to all registered attendees.
29 June, 2026 07:15 am
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08:45 am
Clinical Insights on the Management of NF1-PN Across the Lifespan
Centennial GH
Sponsored by Alexion
7:15 AM: Breakfast service begins
7:30 AM: Program begins
29 June, 2026 08:30 am
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09:00 am
Award Presentation
Centennial DE
2026 CTF Bridge Builder Award:
Presented by Annette Bakker, PhD, Children’s Tumor Foundation and Rosalie Ferner, MD, Guys and St Thomas' Hospital NHS Trust
29 June, 2026 09:00 am
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10:00 am
Keynote: Cancer Immunotherapy Using Non-Viral Genome Engineered Immune Effector Cells
Branden Moriarity PhD
University of Minnesota
Dr. Branden Moriarity is currently an Assistant Professor in the Department of Pediatrics, Division of Hematology/Oncology. He graduated from Saint Olaf College in 2007 with a BA in Biology, Chemistry, and Biomolecular sciences. He received his PhD in Molecular, Cellular, Developmental Biology & Genetics at the University of Minnesota in 2012. From 2012-2014 he was a post doctoral fellow in David Largaespda’s lab, where he worked on identifying the genetics of pediatric sarcomas. He joined the Department of Pediatrics Faculty in 2014.
Dr. Moriarity runs a basic/translational research laboratory working to develop novel cellular therapeutics for gene therapy and cancer immunotherapy with the goal of translating new therapeutics to the clinic. To accomplish these goals, the Moriarity lab uses cutting edge genome engineering technologies, including CRISPR/Cas9, base editor technology, transposons, and rAAV. These tools allow for high frequency gene knockout, gene knock-in, induction of targeted sequence changes, and activation and/or repression of endogenous gene expression. Target cells for engineering include T cells, B cells, NK cells, Monocytes, and hematopoietic stem cells. In addition to developing cellular based therapeutics, the Moriarity lab also performs preclinical drug testing for pediatric cancers, such as osteosarcoma, in order to launch new clinical trials using antibody therapies rather than toxic chemotherapy.
Centennial DE
Branden Moriarity, PhD, University of Minnesota
29 June, 2026 10:00 am
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12:05 pm
Tumor Immunology & Immunotherapy: From Antigens to Adoptive Cell Therapy, Morning Session
David Largaespada
University of Minnesota
Dr. David A. Largaespada is Professor in the Departments of Pediatrics and Genetics, Cell Biology & Development and the Deputy Director of the Masonic Cancer Center at the University of Minnesota, where he also co-directs the Brain Tumor Program and the Center for Genome Engineering. He holds the Hedberg Family/Children’s Cancer Research Fund Chair in Brain Tumor Research. Dr. Largaespada is a leading authority in cancer genetics, functional genomics, and translational modeling, with more than 30 years of continuous research on Neurofibromatosis Type 1 (NF1)–associated tumors. His laboratory has shown that GM-CSF and STAT5 signaling drive juvenile myelomonocytic leukemia (JMML) in NF1, that WNT and SHH signaling define two distinct subtypes of malignant peripheral nerve sheath tumor (MPNST), and that PTEN loss and PI3K activation promote MPNST progression. He has developed novel mouse and human iPSC-based models of plexiform neurofibroma, atypical neurofibroma (ANNUBP), and MPNST, and applied Sleeping Beauty transposon mutagenesis to elucidate the stepwise genetic evolution of these tumors. His group also employs forward genetic and pharmacologic screening to identify synthetic lethal targets and strategies to enhance adoptive cell therapies for cancer. A recipient of the American Cancer Society Research Professor Award, Dr. Largaespada has co-founded four biotechnology companies translating genetic discoveries into clinical innovation.
Nancy Ratner
Cincinnati Children's Hospital
Dr. Ratner is interested in the brain in Neurofibromatosis type 1 and Rasopathies, and in peripheral nerve tumors that occur in the Neurofibromatoses, NF1 and NF2. She uses genomics, animal, and cell culture models to study neurofibroma formation and neurofibroma therapeutics. Ratner received her bachelor's degree from Brown University, her doctorate from Indiana University, Bloomington (during which time she was a student in the Neurobiology Course at MBL), and was a postdoctoral fellow at Washington University in St. Louis. A member of the faculty at the University of Cincinnati from 1987 – 2004, she is currently a Professor in the Department of Pediatrics, Cincinnati Children’s Hospital, University of Cincinnati, and the Program Leader for Cancer Biology and Neural Tumors Program in the Cancer and Blood Disorders Institute where she also co-Leads the Rasopathy Program and holds the Beatrice C. Lampkin Endowed Chair in Cancer Biology. She has served on numerous national and international review panels and authored over 100 peer-reviewed manuscripts and 30 reviews. She was awarded the von Recklinghausen Award in 2010, and received a Jacob K. Javits NIH Neuroscience Investigator Award in 2014.
Steven Rhodes
Dr. Steven Rhodes is an Assistant Professor of Pediatrics at the Indiana University School of Medicine where he directs the Neurofibromatosis (NF) Multidisciplinary Program at the IU Simon Comprehensive Cancer Center. He earned his MD and PhD from the Medical Scientist Training Program at Indiana University, followed by residency training in Pediatrics and fellowship training in Pediatric-Hematology Oncology at Riley Hospital for Children, where he conducted his postdoctoral research in the laboratory of Wade Clapp, MD. Dr. Rhodes has a scientific focus on the development of genetically engineered mouse models that recapitulate the progression of atypical neurofibromas and transformation to malignant peripheral nerve sheath tumor (MPNST) in NF1. Using these models and state-of-the-art approaches in single cell analytics, Dr. Rhodes is working to identify potential therapeutic approaches to both treat and ultimately even prevent MPNST development. Dr. Rhodes’s research is supported by the Francis S. Collins Scholars Program in Neurofibromatosis Clinical and Translational Research funded by the Neurofibromatosis Therapeutic Acceleration Program (NTAP).
Branden Moriarity PhD
University of Minnesota
Dr. Branden Moriarity is currently an Assistant Professor in the Department of Pediatrics, Division of Hematology/Oncology. He graduated from Saint Olaf College in 2007 with a BA in Biology, Chemistry, and Biomolecular sciences. He received his PhD in Molecular, Cellular, Developmental Biology & Genetics at the University of Minnesota in 2012. From 2012-2014 he was a post doctoral fellow in David Largaespda’s lab, where he worked on identifying the genetics of pediatric sarcomas. He joined the Department of Pediatrics Faculty in 2014.
Dr. Moriarity runs a basic/translational research laboratory working to develop novel cellular therapeutics for gene therapy and cancer immunotherapy with the goal of translating new therapeutics to the clinic. To accomplish these goals, the Moriarity lab uses cutting edge genome engineering technologies, including CRISPR/Cas9, base editor technology, transposons, and rAAV. These tools allow for high frequency gene knockout, gene knock-in, induction of targeted sequence changes, and activation and/or repression of endogenous gene expression. Target cells for engineering include T cells, B cells, NK cells, Monocytes, and hematopoietic stem cells. In addition to developing cellular based therapeutics, the Moriarity lab also performs preclinical drug testing for pediatric cancers, such as osteosarcoma, in order to launch new clinical trials using antibody therapies rather than toxic chemotherapy.
Diana Odhiambo
Washington University
Diana Akinyi Odhiambo is an MD–PhD student and emerging physician-scientist dedicated to advancing care and research for patients with neurofibromatosis (NF) and related cancers. Her thesis work sits at the intersection of tumor biology and translational research, with a particular focus on understanding the molecular mechanisms driving tumor progression and therapeutic resistance.
Driven by a deep commitment to impact, Diana approaches science with both passion and vision—connecting bench discoveries to clinical realities. Her research interests include tumor suppressor pathways, genomic instability, the tumor microenvironment, and the unique vulnerabilities of NF-associated tumors, with the goal of identifying targets that can be translated into more effective, patient-centered therapies.
Beyond the lab, Diana is invested in reimagining the future of cancer care globally. Her passion for science and medicine is grounded in a broader commitment to equity, representation, and the expansion of scientific and clinical infrastructure in under-resourced settings.
Lu Le
University of Virginia
Dr. Le is a physician scientist and Kenneth E. Greer, M.D. endowed Professor and Chair of Dermatology Department at the University of Virginia School of Medicine. He received his Ph.D. degree in Immunology and Molecular Genetics, and a medical degree (M.D.) from the Medical Scientist Training Program at the University of California, Los Angeles. He completed an Internship in Internal Medicine at UCLA/St. Mary Medical Center, residency training in Dermatology and a postdoctoral fellowship in Cancer Biology from UT Southwestern Medical Center. His scientific and clinical research focus on neurofibromatosis. A major contribution of Dr. Le’s laboratory has been the generation of novel neurofibromatosis models that decipher mechanisms initiating Schwann cell tumor development and driving their malignant transformation.
Michael Lippincott
University of Colorado Anschutz Medical Campus
Michael J. Lippincott is a PhD candidate at the University of Colorado Anschutz Medical
Campus in the laboratory of Gregory P. Way, PhD. Mike is a computational cell biologist who
utilizes computer vision and data science to derive biological insights from high-dimensional
microscopy image data. He is committed to the mission of reducing suffering for those living with
NF.
Rebecca Dodd
University of Iowa
Rebecca Dodd, PhD, is an Associate Professor of Medicine at the University of Iowa and serves as the Leader of the Cancer Genes and Pathways program at the Holden Comprehensive Cancer Center. She earned her PhD and completed her post-doctoral fellowship at Duke University where she trained in development of preclinical cancer models. Dr. Dodd's translational oncology lab focuses on MPNST biology, metabolism, andmetastasis, with specific interest in tumor evolution, in vivo CRISPR/Cas9 tools, and therapeutic targeting of the tumor microenvironment.
Sarmad Mehmood
University of Alabama
Sarmad Mehmood, PhD, is a Postdoctoral Fellow in the Department of Genetics at the
University of Alabama at Birmingham, where he applies genomic and transcriptomic
approaches to investigate Neurofibromatosis Type 1 (NF1) biology. Under the mentorship
of Dr. Deeann Wallis, he is using single-cell and spatial transcriptomic methods to define
immune dysfunction, checkpoint signaling, and tumor microenvironment remodeling in
NF1-associated tumors. His selected presentation examines conserved NF1-linked
immune evasion programs across NF1 tumors and NF1-deficient cancers, with the goal of
identifying biomarkers and combination strategies relevant to immunotherapy.
Dr. Mehmood’s training spans human molecular genetics, bioinformatics, rare disease
research, and cancer genomics. He earned his PhD in Applied Biosciences from the
National University of Sciences and Technology, Pakistan, and has contributed to gene
discovery and variant characterization in multiple inherited disorders, including
ciliopathies, hereditary neuropathy, muscular dystrophy, and intellectual disability. He has
also contributed to translational cancer genomics research, including studies on
extrachromosomal circular DNA as a potential biomarker.
Through his research, presentations, and collaborative work, Dr. Mehmood is building a
program at the intersection of NF1 biology, cancer immunology, and translational
genomics. He aspires to become a prominent independent investigator in the NF1 field,
with a focus on uncovering disease mechanisms and developing biomarker-driven
therapeutic strategies for patients with NF1-associated and NF1-deficient tumors. In his
free time, he enjoys playing cricket and taking long walks.
Oluwatosin Aina
Medical College of Wisconsin
Oluwatosin Aina is a doctoral student in the Department of Cell Biology, Neurobiology, and Anatomy at the Medical College of Wisconsin (MCW). He is currently mentored by Dr. Daochun Sun. Oluwatosin’s research interests in Neurofibromatosis Type 1 (NF1)-associated tumors include identifying therapeutic strategies, targeting tumor heterogeneity, and remodeling the tumor immune landscape. He currently focuses on repurposing montelukast, an FDA-approved leukotriene receptor antagonist, in combination with selumetinib to overcome adaptive resistance mechanisms and reprogram tumor-associated macrophages in plexiform neurofibromas. His study has been presented at multiple scientific meetings with recognition.
Oluwatosin completed his bachelor's degree with First-Class Honors in Biochemistry from Olabisi Onabanjo University in Nigeria, where he completed a pharmaceutical industry internship, conducted early research in neurotoxicology, and was recognized as the Best Graduating Student in the Faculty of Basic Medical Sciences.
Simeng Lu
Massachusetts General Hospital
Simeng Lu, M.D., is a postdoctoral research fellow at Massachusetts General Hospital and Harvard Medical School. Her current research focuses on NF2-related schwannomatosis, with a particular interest in developing new treatment strategies that can control vestibular schwannoma growth while protecting hearing.
Before joining MGH, Dr. Lu completed five years of residency training in Otolaryngology–Head and Neck Surgery in China. Her earlier research focused on outcomes after cochlear implantation and other approaches to hearing reconstruction.
Through her clinical and research experiences, Dr. Lu has developed a strong commitment to improving care for people with NF2. Her long-term goal is to help translate laboratory discoveries into safer and more effective treatments that preserve hearing and improve quality of life for patients.
Centennial DE
Session Co-Chairs:
Lu Le, MD, PhD, University of Virginia
Nancy Ratner, PhD, Cincinnati Children's Hospital
Lindy Zhang, MD, PhD, Johns Hopkins University
10:00 AM – 10:15 AM: Invited Talk: Tumor Immunology & Immunotherapy: Role of Immune Cells in NF Neoplasms
Lu Le, MD, PhD, University of Virginia
10:15 AM – 10:40 AM: Invited Talk: T Cell Surveillance and Tumor Immune Escape Shape Malignant Outgrowth in NF1 Peripheral Nerve Sheath Tumors
Steven Rhodes, MD, PhD, Indiana University
10:40 AM - 10:55 AM: Break
10:55 AM – 11:20 AM: Invited Talk: Rewiring Tumor Immunity: Sleeping Beauty Screens, Immunopeptidomics, and Tumor Immune Evasion
David Largaespada, PhD, University of Minnesota
11:20 AM - 11:35 AM: Platform Talk: NKG2D Upregulation Enhances T and NK Cell Cytotoxicity, Sensitizes Tumors to Combined αPD1 and αVEGF Therapy, and Contributes to Hearing Loss Prevention in Vestibular Schwannoma Model
Simeng Lu, MD, Massachusetts General Hospital
11:35 AM - 11:50 AM: Platform Talk: Montelukast Suppresses ERBB3-Driven Adaptive Resistance of MEKi and Reprograms Tumor-Associated Macrophages
Oluwatosin Aina, Medical College of Wisconsin
11:50 AM - 12:05 PM: Platform Talk: AXL-CAR-T Therapy as a Potential Treatment for NF1-Related Malignant Peripheral Nerve Sheath Tumor
Ming-Jen Lee, PhD, National Taiwan University Hospital
29 June, 2026 12:05 pm
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01:00 pm
Lunch
Centennial Foyer
Box lunches are available for purchase daily, Saturday through Tuesday. To save time and avoid on-site concession lines, please purchase your box lunch(es) during registration.
29 June, 2026 12:05 pm
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01:00 pm
Variant Curation Expert Panel Meeting (Optional Satellite Meeting)
Centennial GH
Alicia Gomes, MS, CGC, University of Alabama
The neurofibromatosis-schwannomatosis Variant Curation Expert Panel (NF-SWN VCEP) is an international collaboration of researchers, clinicians, and laboratory scientists focused on optimizing the rules for genetic variant classification. This work will help us better determine whether a variant identified during genetic testing for NF or SWN is disease-causing.
This meeting will update attendees on the current classifications regarding NF1 and Legius syndrome. The updated rules will be explained through case examples from the clinic.
This meeting is open to everyone, grab your lunch and join us to learn more.
29 June, 2026 01:00 pm
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04:00 pm
Tumor Immunology & Immunotherapy: From Antigens to Adoptive Cell Therapy, Afternoon Session
Ian Watson
McGill University
Dr. Ian Watson is a member of the Goodman Cancer Institute (GCI), an investigator at the Research Institute of the McGill University Health Centre (MUHC), and an Associate Professor in the Department of Biochemistry at McGill University. He is a Canadian Research Chair II in functional genomics of melanoma, the Co-Chair for the melanoma disease working group for the Canadian Cancer Trials Group (CCTG) and the former Co-Chair for The Cancer Genome Atlas (TCGA) melanoma project. Dr. Watson currently serves as a Steering Committee Member of the Terry Fox Research Institute (TFRI) Marathon of Hope Cancer Centres Network (MOHCCN), a precision oncology initiative with growing international recognition. This network unites clinicians, researchers, and patients across Canada to advance cancer research and care through multi-omic profiling, integrative data-driven research, and the development of learning healthcare systems. He contributed to shaping the program by co-leading early pilot projects that laid the foundation for this $230M national effort. Dr. Watson research program focuses on dissecting how tumor-intrinsic genetic and signaling states in melanoma govern tumor–immune interactions and therapeutic response, leveraging multi-omic and spatial approaches with broad relevance across cancer.
Kimani Njoya
Medical College of Wisconsin
Kimani Njoya is a PhD candidate in the Department of Cell Biology, Neurobiology and Anatomy at the Medical College of Wisconsin in the lab of Dr. Daochun Sun. His research focuses on intercellular signaling and metabolic reprogramming in neurofibromatosis type 1 (NF1)-associated tumors. His scientific training spans experimental and computational approaches across genomics, transcriptomics, and metabolomics at bulk, single-cell, and spatial resolution. He is a recipient of the Children’s Tumor Foundation NF Data Utilization Award, supporting his work integrating lipidomics and transcriptomics to identify targetable metabolic adaptations in NF1 tumors. His first-author study, published in npj Precision Oncology (2025), investigates the natural history of SPP1 signaling across NF1 tumor initiation and progression, and he has co-authored peer-reviewed publications spanning therapeutic strategies for NF1 tumors, vector biology, and computational enhancer prediction.
Centennial DE
Session Co-Chairs:
Lu Le, MD, PhD, University of Virginia
Nancy Ratner, PhD, Cincinnati Children's Hospital
Lindy Zhang, MD, PhD, Johns Hopkins University
1:00 PM - 2:00 PM: Invited Talk: Insights from Transformative Melanoma Therapies: Mechanisms of Response and Implications for NF1-Associated Tumors
Ian Watson, PhD, McGill University
2:00 PM - 2:25 PM: Invited Talk: Rewiring the Immune Landscape of MPNSTs: New Targets and Models for Metastasis and Immunotherapy
Rebecca Dodd, PhD, University of Iowa
2:25 PM - 2:40 PM: Break
2:40 PM - 3:15 PM: Invited Talk: NF-kB pathway inhibition prevents plexiform neurofibroma onset in a mouse model of the disease
Nancy Ratner, PhD, Cincinnati Children’s Hospital Medical Center
3:15 PM - 3:30 PM: Platform Talk: Exploring the interplay between UBR5 expression and NF1-MPNST Immunogenicity: Implications for Immunotherapy
Diana Odhiambo, MD, PhD Trainee, Washington University
3:30 PM - 3:45 PM: Platform Talk: Loss of NF1 drives an immunosuppressive microenvironment and impairs T-cell Function in both neurofibromas and other tumors, providing rationale for targeted ICB therapy
Sarmad Mehmood, PhD, University of Alabama
3:45 PM - 4:00 PM: Platform Talk: Dynamic Remodeling of SPP1-CD44 Signaling Shapes NF1 Tumor Initiation and Progression
Kimani Njoya, Medical College of Wisconsin
29 June, 2026 01:00 pm
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04:00 pm
Brain Development in NF1: New Therapeutic Targets from Bench to Bedside (Concurrent Sesssion)
Jonathan Payne
Murdoch Children’s Research Institute
Dr Payne is a neuropsychologist and clinician-scientist focused on neurodevelopmental outcomes in individuals with NF1. Cognitive, learning, and behavioral difficulties affect the majority of children with NF1 and are among the issues families consistently rank as having the greatest impact on daily life, yet treatment options remain limited. His work aims to change that. He co-leads the Brain & Mind group at the Murdoch Children's Research Institute, is a Senior Neuropsychologist at the Royal Children's Hospital, and is an Honorary Professorial Fellow at the University of Melbourne.
His research program integrates deep behavioral characterization with advanced neuroimaging and preclinical modelling to clarify our understanding of pathways linking genes, molecular signaling, brain, and behavior. He chairs the Medical Advisory Panel for CTF Australia, co-chairs the Neurocognitive Committee of the NF Clinical Trials Consortium, and chairs the Australian Paediatric Neuropsychology Research Network.
Karin S. Walsh
Children’s National Hospital
Karin S. Walsh, Psy.D. is a pediatric neuropsychologist at Children’s National Hospital and Professor of Pediatrics and Psychiatry & Behavioral Medicine at the George Washington University School of Medicine and Health Sciences. A clinician-scientist with over 20 years of experience, Dr. Walsh focuses on neurocognitive outcomes in children with neurofibromatosis type 1 (NF1), other RASopathies, pediatric brain tumors, and hematologic disorders. Her research examines executive function and cerebellar–cortical networks and leads cognitive intervention studies targeting learning and executive dysfunction in pediatric brain tumor and NF1 populations. Dr. Walsh has received multiple competitive research grants and serves in leadership roles across international consortia, including past president of the Posterior Fossa Society and current chair of the Neurocognitive Committee of the NF Clinical Trials Consortium.
D. Wade Clapp
Indiana University
Dr. D. Wade Clapp is the Richard L. Schreiner Distinguished Professor and Chair of Pediatrics at IU School of Medicine, the Associate Dean of Entrepreneurial Research and Commercialization. Dr. Clapp’s laboratory is focused on studying the molecular pathogenesis of genetic disorders with a predisposition to cancer in infants and young children with a particular focus on neurofibromatosis type 1 (NF1). He has been continuously NIH/NCI funded in this area of science for over 25 years. Dr. Clapp has served on a number of national scientific committees in the American Society of Hematology and strategic planning committees for the Children’s Tumor Foundation, which focuses on NF1 and NF2. He also has participated or chaired numerous NIH and DOD peer review panels and been a member of the oversight panel of the DOD NF program. He recently completed a 10 year term as a board member and officer in the American Society of Pediatric Department Chairs (AMSPDC). He continues to lead a physician-scientist pipeline program in AMSPDC charged to increase the number of pediatric physician-scientists in the United States. Dr. Clapp is also active in the NCI-designated comprehensive Indiana University Simon Cancer Center, both as the Chair of the Internal Advisory Board and as a member of the Tumor Microenvironment and Hematopoiesis and Hematologic Malignancies Programs. Dr. Clapp is the Chair of the Biology Committee, and a member of the Plexiform Neurofibroma Committee of the Neurofibromatosis Clinical Trials Consortium.
Dr. Clapp’s laboratory conducts genetic and pharmacologic studies to elucidate key pathological cell- cell and hyperactive Ras-mediated signaling pathways in plexiform neurofibromas using genetically engineered mice developed in his laboratory that closely recapitulate the steps in plexiform neurofibroma formation in humans and the progression of plexiform neurofibromas to atypical plexiform neurofibromas and MPNSTs. Basic work from Dr. Clapp’s laboratory contributed to the concept of haploinsufficiency and the importance of the tumor microenvironment in plexiform neurofibroma initiation and progression. In close collaboration with basic and clinical investigators on project 1, he uses these models as a preclinical platform to inform phase 1-2 trials. These studies include adult and pediatric trials conducted in the DHART SPORE (MPI: Clapp/Shannon) with two mechanistically distinct targeted therapeutics (selumetinib and cabozantinib) that have provided the first broadly successful drugs for plexiform neurofibromas in man.
Erin Gibson
Stanford University
Dr. Gibson is a neuroscientist and Assistant Professor at Stanford University School of Medicine. She received her Bachelors of Science from Duke University and her PhD in from the University of California, Berkeley studying the role of the circadian system in homeostatic processes, including neuroendocrine, immune and neural stem cell regulation. As a postdoctoral scholar at Stanford University, Dr. Gibson studied the effect of neuronal activity on myelin microstructure in health and disease. Dr. Gibson’s lab focuses on understanding how glial cells modulate neural circuits throughout development and in brain disorders, with a focus on the intersection between sleep/circadian and glial biology in disorders such as autism spectrum disorders, multiple sclerosis, and Alzheimer’s disease. By using in vivo mouse and patient-derived iPSC in vitro and ex vivo models, the Gibson lab aims to understand the intrinsic molecular drivers and cell-cell interactions that regulate myelin dynamics throughout life, with the ultimate goal of finding therapeutic strategies to maintain myelin health. She has received numerous awards and honors for her research, including the Chan Zuckerberg Initiative Ben Barres Early Career Accelerator Award, McCormick Award, SRBR Junior Faculty Award, and Psychiatry Innovator Award.
J. Elliott Robinson
Cincinnati Children's Hospital Medical Center
I am a neurobiologist interested in the cognitive symptoms of neurofibromatosis type 1 (NF1) and other Rasopathies, which involve altered cell signaling by the Ras family of proteins. My laboratory in the Rasopathy Program at Cincinnati Children’s Hospital Medical Center (CCHMC) investigates the structure and function of neural circuits involved in motivation, attention, and sensory processing in mouse models of NF1 using cutting-edge systems neuroscience technologies. Additionally, I am working to develop adeno-associated virus (AAV) gene therapies and viral vector-based tools that target signaling pathways dysregulated in NF1. This work is supported by the National Institutes of Health, the Gilbert Family Foundation, the Simons Foundation, and the Neurofibromatosis Therapeutic Acceleration Program. Previously, I received my bachelor’s degree from Georgetown University in 2007, followed by my MD and PhD from the Medical Scientist Training Program at the University of North Carolina at Chapel Hill in 2016. I was a post-doctoral fellow in the laboratory of Dr. Viviana Gradinaru at Caltech from 2016-2020, where I studied dopaminergic circuit dysfunction in NF1. I joined the Division of Experimental Hematology and Cancer Biology at CCHMC in 2020.
Sattar Khoshkhoo
Brigham and Women's Hospital, Harvard Medical School
Dr. Khoshkhoo is a physician-scientist and Director of the Epilepsy Genetics Program at Mass General Brigham and Harvard Medical School. He completed medical training at UCSF, adult neurology residency at Mass General Brigham, and clinical epilepsy training at Brigham and Women’s Hospital and Boston Children’s Hospital. After his medical training and as a research fellow at Boston Children’s Hospital he shifted his research focus to the genetic underpinnings of focal epilepsies, which he continues to work on in has lab today. Dr. Khoshkhoo’s lab is broadly interested in understanding the role of genetic mosaicism in normal development and in neurological diseases with a special focus on RASopathies.
Shruti Garg
University of Manchester
Dr Shruti Garg is a Clinical Senior Lecturer in Translational Child and Adolescent Psychiatry at the University of Manchester and Honorary Consultant at Royal Manchester Children's Hospital. Her research focuses on NF1 and related RASopathies, using neuroimaging, MR spectroscopy, EEG, and non-invasive brain stimulation to investigate the brain mechanisms underlying autism, ADHD, and cognitive difficulties, and to test new interventions from infancy through adolescence. Her population-based studies were among the first to establish the link between NF1 and autism. In 2018, she was awarded the Francis Collins Scholarship by the Neurofibromatosis Therapeutic Acceleration Programme at Johns Hopkins University. Her research is currently funded by the MRC, NIHR Biomedical Research Centre, and US Department of Defense. She serves on the medical advisory boards of Nerve Tumours UK, and the European Children's Tumor Foundation.
Tamar Green
Stanford University
Dr. Tamar Green is a physician-scientist dedicated to translating genetic discoveries into more effective treatments for children with neurodevelopmental disorders. She earned her MD from Ben-Gurion University and completed her training in child psychiatry at Tel Aviv University. Following that, she pursued postdoctoral research in neuroscience at Stanford University. She now serves as an Associate Professor in the Department of Psychiatry and Behavioral Sciences at Stanford, where she leads the Brain Imaging, Development, and Genetics (BRIDGE) Lab.
Her research is grounded in a genetic first approach. By starting with clearly defined genetic mutations, she investigates how these alterations influence brain development and behavior. This strategy allows her to trace specific biological pathways and connect them directly to clinical outcomes that matter for patients and families.
Dr. Green's work focuses on rare genetic conditions, including 22q11.2 deletion syndrome, Turner syndrome, and more recently, RASopathies such as Noonan syndrome and Neurofibromatosis 1. She brings together genetics, advanced brain imaging, and clinical insight to reveal how these mutations impact brain structure and function.
Dr. Green’s work moves beyond identifying mechanisms to testing how those insights can inform treatment. Her team aims to study targeted therapies like MEK inhibitors, with the goal of evaluating how these interventions affect the brain and cognition in real-world settings. By identifying reliable brain-based markers of treatment response, her research moves the field closer to precision medicine. Backed by NIH funding and recognized with honors such as the Francis S. Collins Scholar Award, Dr. Green is helping to shift the focus from managing symptoms to modifying the developmental course of these complex disorders.
Yang Hou
College of Medicine at Florida State University
Dr. Yang Hou is an Assistant Professor and Director of the Development, Environment, and Resilience (DEaR) Lab in the College of Medicine at Florida State University. She earned her Ph.D. in Human Development and Family Sciences from the University of Texas at Austin and completed postdoctoral training in health psychology and neurobehavioral research at the National Cancer Institute.
Dr. Hou’s research examines biopsychosocial influences on neurobehavioral development—including cognitive, academic, socioemotional, and behavioral functioning—among underrepresented populations, particularly individuals with rare genetic conditions such as neurofibromatosis type 1 (NF1). Her work leverages innovative and advanced quantitative methods to uncover patterns and predictors of neurobehavioral development across the lifespan. Her long-term goal is to inform scalable, data-driven interventions that improve neurobehavioral outcomes in individuals with NF1 and other underrepresented populations.
In addition to leading her research program, Dr. Hou contributes as a biostatistician on interdisciplinary teams and is committed to advancing developmental and clinical science through rigorous methodology, open science practices, and data sharing.
Dr. Hou has published over 60 peer-reviewed articles, and her research has been supported by the U.S. Department of Defense and the Children’s Tumor Foundation. She has received international recognition for her contributions, including the Rising Star Award from the Association for Psychological Science and the Early Career Outstanding Paper Award from the American Psychological Association. She currently serves as an Associate Editor for the Journal of Research on Adolescence.
Zoe Cappel
Cincinnati Children's Hospital Medical Center
I am a neuroscientist interested in the cell lineages and brain circuits that drive cognitive dysfunction and attentional phenotypes in Neurofibromatosis Type 1 (NF1) and other Rasopathies. I am a senior neuroscience graduate student at the University of Cincinnati, working in the lab of Dr. J. Elliott Robinson at Cincinnati Children’s Hospital Medical Center. I was recently awarded the Children’s Tumor Foundation Young Investigator Award in 2025 to support my project, which utilizes a novel transgenic approach and cutting-edge systems neuroscience technologies to investigate how altered Ras-MAPK activity affects the function of brain circuits to produce behavioral changes. Previously, I earned my bachelor’s degree in molecular biology from DePauw University, where I was a Science Research Fellow and gained research experience in biochemistry, biology, and neuroscience labs.
Centennial GH
Session Co-Chairs:
D. Wade Clapp, MD, Indiana University
Tamar Green, MD, Stanford University
1:00 PM - 1:05 PM: Welcome and Introduction
D. Wade Clapp, MD, Indiana University
1:05 PM - 1:35 PM: Invited Talk: From Brain Wiring to Behavior: White Matter Findings in NF1 and Related Conditions
Tamar Green, MD, Stanford University
1:35 PM - 1:55 PM: Invited Talk: Contribution of Ras-MAPK Mosaicism in Drug-Resistant Epilepsy and Therapeutic Opportunities
Sattar Khoshkhoo, MD, Brigham and Women's Hospital, Harvard Medical School
1:55 PM - 2:15 PM: Invited Talk: A Novel Ex Vivo Platform for Studying Human Myelination
Erin Gibson, PhD, Stanford University
2:15 PM - 2:35 PM - Break
2:35 PM - 2:50 PM: Platform Talk: Cortical Activation of the Ras-MAPK Pathway Reproduces Neurological Deficits Seen in NF1 Model Mice
Zoe Cappel, Cincinnati Children's Hospital Medical Center
2:50 PM - 3:05 PM: Platform Talk: Distinct Neuropsychological Subgroups in Pediatric Neurofibromatosis Type 1 and Their Correlates: A Multi-Center Study
Yang Hou, PhD, College of Medicine at Florida State University
3:05 PM - 3:50 PM: Interactive Expert Panel: Translating Research into Clinical Practice
Moderators:
Wade Clapp, MD
Tamar Green, MD
Panelists:
Shruti Garg, MD, University of Manchester
Erin Gibson, PhD, Stanford University
Sattar Khoshkhoo, MD, Brigham and Women's Hospital, Harvard Medical School
Jonathan Payne, PsyD, Murdoch Children's Research Institute
Elliott Robinson, MD, PhD, Cincinnati Children's Hospital Medical Center
Karin Walsh, PsyD, Children's National Hospital
3:50 PM - 4:00 PM: Closing Remarks and Collaborative Opportunities
Tamar Green, MD
29 June, 2026 04:00 pm
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06:35 pm
Clinical Platform Session
Matthias Karajannis
Memorial Sloan Kettering
Dr. Matthias Karajannis has been serving as the Chief of the Pediatric Neuro-Oncology Service at Memorial Sloan Kettering Cancer Center since 2017. He is a Member of the Memorial Hospital faculty and Professor of Pediatrics at Weill Cornell Medical College in New York. Previously, he directed the NF Clinical Research Program and the Pediatric Hematology/Oncology Fellowship Training Program at NYU Langone Medical Center. He received his MD from the Free University Berlin, Germany, completed residency training at Duke University Medical Center, and fellowships in pediatric hematology/oncology, as well as pediatric neuro-oncology, at Memorial Sloan Kettering Cancer Center. Dr. Karajannis treats children and young adults with tumors of the nervous system, and has a special interest in caring for patients with NF2-SWN. His research aims at developing novel diagnostic tools and molecular targeted therapies for patients with nervous system tumors, including NF2-SWN patients, through translational research and clinical trials. Dr. Karajannis serves as principal investigator and study chair for cooperative group clinical trials with the Children’s Oncology Group (COG), Pediatric Brain Tumor Consortium (PBTC) and NF Clinical Trials Consortium (NFCTC), where he chairs a recently completed phase 2 study for patients with NF2-SWN and progressive vestibular schwannoma.
Nancy Ratner
Cincinnati Children's Hospital
Dr. Ratner is interested in the brain in Neurofibromatosis type 1 and Rasopathies, and in peripheral nerve tumors that occur in the Neurofibromatoses, NF1 and NF2. She uses genomics, animal, and cell culture models to study neurofibroma formation and neurofibroma therapeutics. Ratner received her bachelor's degree from Brown University, her doctorate from Indiana University, Bloomington (during which time she was a student in the Neurobiology Course at MBL), and was a postdoctoral fellow at Washington University in St. Louis. A member of the faculty at the University of Cincinnati from 1987 – 2004, she is currently a Professor in the Department of Pediatrics, Cincinnati Children’s Hospital, University of Cincinnati, and the Program Leader for Cancer Biology and Neural Tumors Program in the Cancer and Blood Disorders Institute where she also co-Leads the Rasopathy Program and holds the Beatrice C. Lampkin Endowed Chair in Cancer Biology. She has served on numerous national and international review panels and authored over 100 peer-reviewed manuscripts and 30 reviews. She was awarded the von Recklinghausen Award in 2010, and received a Jacob K. Javits NIH Neuroscience Investigator Award in 2014.
Joanna Lempiainen
Washington University School of Medicine,
Dr. Joanna Lempiäinen is a postdoctoral research associate in the laboratory of Dr. Benjamin
Garcia at Washington University School of Medicine in St. Louis, where she studies epigenetic
dysregulation in malignant peripheral nerve sheath tumors (MPNSTs). Her work integrates
quantitative mass spectrometry with genome-wide sequencing approaches to understand how
chromatin alterations drive tumor biology.
Dr. Lempiäinen completed her Ph.D. at the University of Eastern Finland in the laboratory of Dr.
Jorma Palvimo, where she investigated protein-protein interactions of steroid hormone
receptors on chromatin. During her doctoral studies, she applied proximity-labeling and
chromatin proteomics approaches to uncover novel regulatory interactions and validated these
findings using ChIP-seq, RNA-seq, and functional perturbation in prostate cancer models.
In her postdoctoral research, Dr. Lempiäinen has expanded her expertise to mass spectrometry-
based quantitative proteomics, including profiling of total proteomes and histone post-
translational modifications. She applies these approaches to cancer epigenetics, focusing on
the molecular consequences of PRC2 loss in MPNST. Her work integrates proteomic,
epigenomic, and transcriptomic data to define epigenetically driven oncogenic programs,
including dysregulation of IGF2 signaling.
Dr. Lempiäinen’s long-term goal is to establish an independent research program focused on
chromatin-based mechanisms in cancer and to develop therapeutic strategies targeting
epigenetic vulnerabilities.
Lauren Weintraub
Albany Medical Center
Lauren Weintraub, MD, is the director of the Pediatric Brain Tumor and Neurofibromatosis Program at Albany Medical Center. She specializes in diagnosing and treating infants, children, and adolescents with a wide range of childhood cancers and blood disorders. Dr. Weintraub also serves as a Professor of Pediatrics at Albany Medical College. Dr. Weintraub attended Tufts University for medical school. She completed her residency in pediatrics at Icahn School of Medicine at Mount Sinai Hospital and a fellowship in pediatric hematology and oncology at Children’s Hospital at Montefiore.
Stephen Gilene
Cincinnati Children's Hospital Medical Center
I received my medical degree from Boonshoft School of Medicine prior to completing both a pediatric residency and hematology/oncology fellowship at Cincinnati Children’s Hospital Medical Center (CCHMC). As a newly appointed faculty member in the Cancer and Blood Diseases Institute at CCHMC, my focus lies in pediatric solid tumor malignancies. I have a special interest in neurofibromatosis type I stemming from years of training in Nancy Ratner’s lab during fellowship. There I studied the biology and treatment of neurofibromatosis type 1 associated plexiform neurofibromas. This included investigation into the development of gene therapies for rescuing neurofibromin function through adeno-associated viral vectors in murine models as well as identification of circulating plasma biomarkers for plexiform neurofibroma tumor burden and treatment response. This interest has extended into the clinical domain where I am a member of the Neurofibromatosis Clinic responsible for the comprehensive, multidisciplinary care of patients with these disorders.
Xiyuan Zhang
Rutgers University
Dr. Xiyuan Zhang is an Assistant Professor in the Department of Pediatrics, Division of Pediatric Hematology/Oncology, at Robert Wood Johnson Medical School of Rutgers University. She is also a faculty member at the Rutgers Cancer Institute. She is leading the Molecular Cancer Prevention Laboratory at Rutgers in New Jersey. The work in her lab leverages genome-wide sequencing technologies to decipher the etiology and progression of NF1-associated nerve sheath tumors. Their work is dedicated to enhancing the diagnostic, preventive, and therapeutic utilities of molecular profiling, aiming to improve our understanding of cancer biology and benefit our patients with targeted therapies.
Zhichao Wang
Shanghai Jiao Tong University School of Medicine
Prof./Dr. Zhichao Wang is a Chief Surgeon and Associate Professor in the Department of Plastic and Reconstructive Surgery at Shanghai Ninth People’s Hospital, Shanghai Jiao Tong University School of Medicine, and serves as a PhD supervisor. He obtained his MD from Shanghai Medical College of Fudan University and completed an MPH in Clinical Trials and Biostatistics at the Johns Hopkins Bloomberg School of Public Health.
He has dedicated his career to the clinical and scientific research of NF1. He serves as PI and leads the MDT for comprehensive NF1 management at Shanghai Ninth People’s Hospital, with over 3,500 outpatient visits and 500+ surgeries annually. His team has established the first and largest NF1 biobank and clinical cohort in China, the CHINA NICE COHORT (China Neurofibromatosis Integrated Clinical and Epidemiological Cohort). Based on this platform, he led the first Phase I clinical trial of selumetinib in pediatric NF1 patients in China, and has conducted multiple Phase I and Phase IIa clinical studies of MEK inhibitors in adult patients with PNF, including selumetinib and two novel agents, HL-085 and TQ-B3234.
To date, Dr. Wang has published 57 papers in the NF1 field as first or corresponding author in journals such as The BMJ, Clinical Cancer Research, npj Digital Medicine, Advanced Science, JAMA Dermatology, International Journal of Surgery, Signal Transduction and Targeted Therapy, and Journal of Investigative Dermatology (with accompanying Editorials). He was also invited to contribute a Correspondence to The Lancet Neurology, where he systematically introduced China's experience in the holistic management and cohort establishment of NF1.
In recognition of these contributions, Dr. Wang serves as the lead author and Vice Chair of the national Neurofibromatosis Study Group under the Chinese Society of Plastic Surgery, where he spearheaded the development of 2 national expert consensus statements on NF1. He has also been selected as a member of the Asia-Pacific NF1 Expert Consensus Steering Committee.
Lei Xu
Massachusetts General Hospital
Dr. Lei Xu is an Associate Professor of Radiation Oncology at Massachusetts General Hospital and Harvard Medical School. Xu’s lab has established patient-derived preclinical models that recapitulate the neurological deficits associated with NF2-related, as well as non-NF2 related schwannomatosis. Using these tools, research in the Xu lab focuses on characterizing the tumor microenvironment and investigating its role in disease progression and therapy resistance. Her work is supported by the NIH, Children’s Tumor Foundation, and American Cancer Society.
Heather Radtke
Medical College of Wisconsin
Eva Dombi
National Cancer Institute
Dr. Dombi received her M.D. at the Semmelweis University, Budapest, Hungary. She completed her pediatric residency training in Budapest, Hungary, prior to moving with her family to the United States. She has been a PET Section member since 2002. Dr. Dombi leads the PET Section Imaging Program. One of her many roles has been the support of research efforts related to the development of novel medical treatments for children and young adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas. Dr. Dombi had a critical role in the development and validation of a novel method of image analysis for these tumors, which is now used in most ongoing clinical trials nationwide to assess the primary endpoint of disease progression. Dr. Dombi performs central response evaluation of plexiform neurofibromas using volumetric MRI on multiple clinical trials. In addition, Dr. Dombi has several ongoing collaborations with extramural investigators.
Ayo Adeyemi
Alexion Pharmaceuticals
Ayo Adeyemi is Director of Global Health Economics and Outcomes Research (HEOR) at Alexion, AstraZeneca’s Rare Disease Unit, based in Boston. She leads the development of evidence strategies to demonstrate the value of innovative therapies, with a particular focus on rare diseases and oncology.
Ayo has extensive experience in designing and delivering patient-centered evidence, including patient-reported outcomes, real-world evidence, and health technology assessment support. Her work emphasizes translating complex clinical and economic data into impactful insights that inform decision-making across stakeholders.
She has been actively involved in advancing evidence generation for conditions such as plexiform neurofibromas (PN), collaborating cross-functionally and with external partners to shape research, and access strategies.
Ayo is passionate about improving outcomes for patients through rigorous evidence and meaningful stakeholder engagement, and she is a regular contributor to scientific discussions and conferences in the field.
Mayowa Osundiji
Mayo Clinic
Dr. Mayowa Osundiji is a board-certified Medical Geneticist and Molecular Diagnostics specialist at Mayo Clinic Arizona, where he serves as Director of the Children’s Tumor Foundation Neurofibromatosis and Schwannomatosis Clinic. He has extensive expertise in the diagnosis and management of genetic disorders across the lifespan, with a particular focus on cancer genetics, vascular, musculoskeletal, and neurogenetic conditions. Dr. Osundiji completed his Ph.D. in Medicine at the University of Cambridge and his medical training at St. Bartholomew’s Hospital and The Royal London Hospital, University of London. He underwent specialty training in Medical Genetics at Mayo Clinic and completed a postdoctoral research fellowship at Dana-Farber Cancer Institute, Harvard Medical School. Dr. Osundiji's work bridges clinical medicine and translational science, with research interests centered on systems biology, nutrient metabolism, and the molecular determinants of cellular survival and tissue integrity in human disease. He currently serves as Chair of the Treatment Guidelines Working Group for the National Organization for Rare Disorders and is a member of the ClinGen Urea Cycle Disorders Variant Curation Expert Panel.
Centennial DE
Session Co-Chairs:
Andrea Gross, MD, Cincinnati Children’s Hospital Medical Center
Zhichao Wang, MD, PhD, Shanghai Jiao Tong University School of Medicine
4:00 PM - 4:05 PM: Welcome and Introduction
Andrea Gross, MD, Cincinnati Children’s Hospital Medical Center
Zhichao Wang, MD, PhD, Shanghai Jiao Tong University School of Medicine
4:05 PM - 4:20 PM: Long-Term Analysis of Mirdametinib Treatment in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma (NF1-PN): Updated Phase 2b ReNeu Trial Findings
Angela Hirbe, MD, PhD, Washington University School of Medicine
4:20 PM - 4:35 PM: Impact of selumetinib on pain in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (NF1-PN): an in-depth analysis from the international, phase 3, randomized, placebo-controlled KOMET trial
Ayo Adeyemi, Alexion Pharmaceuticals
4:35 PM - 4:50 PM: Pain Outcomes in Patients With and Without Volumetric Tumor Response: Post Hoc Analysis From the ReNeu Trial of Mirdametinib in Adults and Children With Neurofibromatosis Type 1-Associated Plexiform Neurofibroma (NF1-PN)
Lauren Weintraub, MD, Albany Medical Center
4:50 PM - 5:05 PM: Plasma Biomarker Profiling in Neurofibromatosis Type 1 Associated Plexiform Neurofibroma. Summary: Evaluation of plasma biomarkers in relationship to neurofibromatosis type 1 (NF1) associated plexiform neurofibroma (PNF) with cohorts including healthy controls, NF1 without PNF, NF1 with PNF before and during MEK inhibition and a PNF murine model.
Stephen Gilene, MD, Cincinnati Children's Hospital Medical Center
5:05 PM - 5:20 PM: cNF-Vision: Lesion-Level Image Analysis of Cutaneous Neurofibromas in a large Neurofibromatosis Type 1 Cohort
Christoph Sadée, MS, Stanford University
5:20 PM - 5:35 PM: Break
5:35 PM - 5:50 PM: Initial Findings of the NIH Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST) Observational Study
Eva Dombi, MD, National Cancer Institute
5:50 PM - 6:05 PM: NF110: A Neurofibromatosis Clinical Trials Consortium Phase 2 Clinical Trial of Crizotinib for Children and Adults with NF2-related Schwannomatosis and Progressive Vestibular Schwannomas
Matthias Karajannis, MD, Memorial Sloan Kettering
6:05 PM - 6:20 PM: 12 years of LZTR1-related Disorders: Expanding the Phenotype and Genotype Spectrum with Context Specificity
Mayo Osundiji, PhD, Mayo Clinic
6:20 PM - 6:35 PM: International Approaches to Neurofibromatosis and Schwannomatosis Guidelines: The Need for Genetic Testing Recommendations
Heather Radtke, MS, CGC, Medical College of Wisconsin
29 June, 2026 04:00 pm
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06:35 pm
Basic / Preclinical Platform Session (Concurrent Session)
Harrison Parent
Vanderbilt University
Harrison received his B.S. in Biochemistry and Molecular/cellular biology for the University of New Hampshire in 2022 before beginning his PhD studies at Vanderbilt University later that year. Harrison joined the lab of Dr. Colleen Niswender in the Department of Pharmacology; a group specializing in neuroscience drug discovery efforts concerning the metabotropic glutamate receptor family. Harrison’s project has identified a novel correlation between metabotropic glutamate receptor 7 (mGlu7) and NF1, and is currently focused on investigating the mechanisms underlying this genetic association and the potential of mGlu7 as a modifier for cognitive NF1 phenotypes. Using a variety of techniques spanning molecular pharmacology, electrophysiology, behavioral pharmacology, and human genetic data, the goal of this project is to lay the groundwork for new drug discovery and development efforts into mGlu7. Ultimately, Harrison hopes to provide a novel approach to treat cognitive impairments and learning disabilities associated with NF1, which currently lack effective therapeutic options.
Kyle Williams
University of Minnesota Twin Cities
Kyle B. Williams, Ph.D. is an Assistant Professor in the Division of Pediatric Hematology/Oncology at the University of Minnesota. Dr. Williams's laboratory is dedicated to developing new and effective treatments for pediatric solid tumors, with a particular focus on neurofibromatosis type 1 (NF1). As a postdoctoral fellow in the laboratory of Dr. David Largaespada at the University of Minnesota, he honed his expertise in genome engineering to investigate NF1 biology and drug susceptibility, work that continues to drive his current research program. Dr. Williams is a former Children's Tumor Foundation Young Investigator, Children's Cancer Research Fund Emerging Scientist, and recent recipient of the NTAP Basic and Translational Laboratory Scholars (BTLS) Award. By leveraging advanced genome engineering and genetic screening technologies, his laboratory works to accelerate the discovery and preclinical development of promising new small molecule and cellular immunotherapies for children with cancer. This work is highly collaborative with the NF clinical team at the University of Minnesota, a partnership that supports ongoing clinical trials for NF1 and has opened a new clinical trial for the treatment of malignant peripheral nerve sheath tumors (MPNST).
Ethan Hass
University of Central Florida
My interest in neuroscience and glial cell biology started as an undergraduate student at the University of Florida under Dr. Benoit Giasson, Ph.D. and M.D./Ph.D. student Zachary Sorrentino. There, I conducted guided research on neurodegenerative diseases, specifically on mouse models of synucleinopathy. As part of my undergraduate thesis, I investigated oligodendrocyte α-synuclein inclusion pathology in multiple system atrophy with patient tissues generously provided by the McKnight Brain Institute. It was in this lab that I developed a deep passion for uncovering molecular events that lead to devastating diseases and the value my research can have on those afflicted with them.
After graduation, I started my career in medicine as part of the M.D./Ph.D. program at the University of Central Florida and found a home in the laboratory of Dr. Cristina Fernandez-Valle, where I now conduct high-throughput and targeted drug efficacy studies on patient-derived schwannoma cells. For my doctoral research, I published three original works: one characterizing primary schwannomatosis patient-derived cells and their drug sensitivities, one elucidating the mechanism by which HDAC inhibition promotes cell death in schwannoma cells, and a brief communication exploring potential alternative therapies for malignant peripheral nerve sheath tumors. I find satisfaction in knowing the work I do is for the betterment of others’ lives, especially those affected by serious rare tumors. My work emphasizes the importance of translational cell-based studies on uncovering novel therapeutic approaches for patients with schwannomatosis and neurofibromatosis.
Kimia Rayat Sanati
The University of Alabama at Birmingham
Kimia is a second-year doctoral trainee in the Graduate Biomedical Sciences (GBS) program at the University of Alabama at Birmingham (UAB), training within the Pathobiology, Pharmacology, and Physiology theme. Working in the laboratory of Dr. Deeann Wallis in the Department of Genetics, her research investigates the molecular basis of Neurofibromatosis Type 1 with a focus on developing targeted therapeutics.
Her work centers on advancing antisense oligonucleotide (ASO)-based therapies for NF1, including exon skipping and cryptic splice site repression, by addressing two critical gaps in the field: the limitations of current ASO delivery systems and the absence of precision preclinical models for NF1. To that end, she systematically evaluates ASO therapies delivered via AAVs and peptide-conjugated PMOs across three complementary novel mouse models, with the goal of bringing these approaches closer to clinical reality for patients with NF1.
Kimia is a recipient of the HSOM STAR21 Award, a competitive scholarship from the Marnix E. Heersink School of Medicine at UAB, granted in recognition of her doctoral research potential. She is honored to present her work at the NF 2026 conference.
Belinda Garana
Sage Bionetworks
elinda Garana, PhD is a Senior Biomedical Data Manager at Sage Bionetworks. Previously, Dr. Garana researched NF1-MPNST and other cancers as a Post Doctorate Research Associate in computational biology at the Pacific Northwest National Laboratory. She earned her PhD in chemical engineering in 2023 from the University of Southern California and her BS in 2018 from the University of Washington.
Juri Na
University of Plymouth
Dr Juri Na is a molecular biologist and senior research fellow at the University of Plymouth, based in the Brain Tumour Research Centre of Excellence. Her research focuses on improving treatment strategies for meningioma, with a particular emphasis on enhancing radiotherapy efficacy and overcoming resistance. Her work combines molecular and cellular approaches to identify novel radiosensitisers, particularly through the repurposing of clinically approved drugs. She has expertise in high-throughput drug screening, cell cycle analysis, and DNA damage response, and develops advanced in vitro models to study treatment responses. Dr Na has published in peer-reviewed journals on the use of histone deacetylase inhibitors as radiosensitisers in meningioma. Her current research aims to translate laboratory findings into clinically relevant strategies to improve outcomes for patients with brain tumours. Alongside her research, she is actively involved in student supervision and laboratory training.
Centennial GH
Session Co-Chairs:
Juri Na, PhD, University of Plymouth
Xiyuan Zhang, PhD, Rutgers University
4:00 PM - 4:05 PM: Welcome Remarks
Juri Na, PhD, University of Plymouth
Xiyuan Zhang, PhD, Rutgers University
4:05 PM - 4:20 PM: KRAS is required for plexiform neurofibroma formation and is a targetable vulnerability in established Nf1-deficient neurofibromas
Nancy Ratner, PhD, Cincinnati Children's Hospital Medical Center
4:20 PM - 4:35 PM: Epigenomic Rewiring in PRC2-Deficient Malignant Peripheral Nerve Sheath Tumors Uncovers an IGF2-Driven Therapeutic Vulnerability
Joanna K. Lempiainen, PhD, Washington University School of Medicine
4:35 PM - 4:50 PM: Combined MEK and HDAC Inhibition in PRC2-Deficient MPNST: Preclinical Validation and Initial Clinical Experience from a Phase 0 “Window of Opportunity” Trial
Kyle Williams, PhD, University of Minnesota Twin Cities
4:50 PM - 5:05 PM Encapsulating CUDC-907 in Controlled-release Nanoparticles Increases Its Potency in NF2-related Human Schwannoma Cells
Ethan Hass, BS, University of Central Florida
5:05 PM - 5:20 PM: A cochlea-sparing strategy for non-invasive control of intracranial schwannomas via peripheral irradiation and anti-PD-1 therapy enhanced by STING activation
Lei Xu, MD, Massachusetts General Hospital
5:20 PM - 5:35 PM: Break
5:35 PM - 5:50 PM: Targeting Neuronal Signaling via Metabotropic Glutamate Receptor 7 (mGlu7) to Treat Neurofibromatosis Type 1 (NF1)-Related Cognitive Impairment
Harrison Parent, Vanderbilt University
5:50 PM - 6:05 PM: Restoration of neurofibromin expression by masking the recurrent pathogenic variant c.1466A>G (p.Y489C) using AAV-F delivery of U7-SnRNA constructs in a preclinical mouse model of Neurofibromatosis type 1
Kimia Rayat Sanati, University of Alabama
6:05 PM - 6:20 PM: ZNF423 defines a therapeutically exploitable transcriptional dependency in NF1-associated MPNST
Sarah Morrow, Indiana University
6:20 PM - 6:35 PM: Young Investigator Day Winner
30 June, 2026 07:00 am
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08:30 am
Breakfast
Centennial Foyer
Light breakfast is available to all registered attendees.
30 June, 2026 08:00 am
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09:00 am
Poster Competition Finalists: Platform Presentations
Centennial DE
Finalists will give a platform presentation with time for Q&A
30 June, 2026 09:00 am
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10:00 am
Keynote: Strategies for Selective Anticancer Drug Discovery
Paul J Hergenrother
University of Illinois
Paul J. Hergenrother was born in 1972 and raised in Akron, Ohio. He attended the University of Notre Dame, where he received his B.S. in Chemistry in 1994. From there Paul moved to the University of Texas at Austin to conduct graduate research under the direction of Professor Stephen F. Martin, and moved on as an American Cancer Society postdoctoral fellow to Harvard University, where he worked in the laboratory of Professor Stuart L. Schreiber.
He established his own laboratory in the Department of Chemistry at the University of Illinois at Urbana-Champaign in 2001, and in 2013 was named the Kenneth L. Rinehart Endowed Chair in Natural Products Chemistry. Professor Hergenrother is the co-founder and Chief Scientific Officer of Vanquish Oncology, and through Vanquish an anticancer compound discovered by the Hergenrother lab (the procaspase-3 activator PAC-1) completed a Phase 1 clinical trial in late-stage cancer patients, and has also been assessed in Phase 1b/2 trials in glioblastoma and metastatic uveal melanoma. The Hergenrother lab, in collaboration with the laboratory of Prof David Shapiro (UIUC Biochemistry), also discovered the anticancer compound ErSO, with potent activity against ER+ breast tumors; ErSO was licensed to Systems Oncology who subsequently licensed it to Bayer. More recently, next-generation ErSO derivatives were licensed to Oncoteq.
In spring of 2020, while UIUC was shut down due to the COVID-19 pandemic, Prof. Hergenrother led the team that developed a novel saliva-based assay for SARS-CoV-2, a simple protocol that bypasses the need for RNA isolation and as such is inexpensive, returns rapid results, and can be used on scale. The University of Illinois administered over 1,000,000 of these tests on its campus during Fall 2020, typically testing ~10,000 people a day. With Emergency Use Authorization (EUA) status, over 120 colleges, universities, schools, and businesses used this test.
Most recently he was named as the recipient of the 2016 UCB-Ehrlich Award for Excellence in Medicinal Chemistry, of the 2016 Akron Section Award from the ACS, a 2017 ACS Arthur C. Cope Scholar Award, and the 2018 ACS Sosnovsky Award for Cancer Research. He was named as a Fellow of the National Academy of Inventors, and in 2023 he received the Outstanding Investigator Award from the National Cancer Institute.
At the University of Illinois Professor Hergenrother is the Leader of the IGB Theme “Anticancer Discovery from Pets to People”, is the Director of the NIH Chemistry-Biology Interface Training Grant, and is Deputy Director of the Cancer Center at Illinois.
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Paul J. Hergenrother, PhD, University of Illinois
30 June, 2026 10:00 am
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12:25 pm
NextGen Translational Pipelines: From Models to Clinical Impact, Morning Session
Laura Klesse
UT Southwestern
Dr Laura Klesse is a pediatric neuro-oncologist who specializes in the care of patients with neurofibromatosis and central nervous system tumors. Dr. Klesse is currently the Director of the Comprehensive Neurofibromatosis and Associate Division Chief of Research Operations at UT Southwestern and Children’s Health in Dallas, Texas. She serves as the site’s principal investigator for the National NF Clinical Trials Consortium, the Children’s Oncology Group and as chair of the bone subcommittee for the NFCTC. Dr Klesse is nationally recognized as a clinical leader in NF patient care and is currently Chair of the Children’s Tumor Foundation’s Clinical Care Advisory Board. Dr Klesse is an Professor of Pediatrics and Neurological Surgery at UT Southwestern and is a Dedman Family Scholar in Clinical Care.
Rosalie Ferner
Guys and St Thomas' Hospital NHS Trust
Rosalie Ferner is an adult neurologist at Guy’s and St. Thomas’s NHS Foundation Trust. She was national lead clinician for the nationally commissioned Complex Neurofibromatosis 1 (NF1) Service from the launch in 2009 until 2022; she was the London lead for the national NF2 service from 2010-2014. She is a Trustee and member of the Medical Advisory Board for the patient organisation Nerve Tumours UK. She is a recipient of the European Theodor Schwann award (2016) and the von Recklinghausen award (2024).
She has extensive clinical and research experience in neurofibromatosis and schwannomatosis. The Neurofibromatosis Centre at Guy’s Hospital has about 2,000 patients and both children and adults are seen in the same department. The aim of the national NF1 service is to provide expert, lifelong, multi-disciplinary, holistic care for people with complex, multi-system disease. A transition service has been developed and expanded since 2009.
Rosalie Ferner’s doctoral thesis was on “Intellectual problems in neurofibromatosis 1” and her research interests include defining clinical phenotype and natural history in NF1, diagnosis of malignant peripheral nerve sheath tumours in NF1, and the development of robust clinical and patient focused outcome measures for monitoring therapy in NF1 and NF2 schwannomatosis.
Disclosures
Rosalie Ferner has acted as medical adviser on neurofibromatosis 1 for Astra Zeneca and Alexion and participated in educational sessions
Tena Rosser
Children’s Hospital of Los Angeles
Dr. Tena Rosser is an Associate Professor of Pediatrics and Neurology at the Children’s Hospital Los Angeles which is affiliated with the USC Keck School of Medicine. She is the director of the CHLA Children’s Tumor Foundation-endorsed Neurofibromatosis Clinic which opened in 2005. She is also a member of the Children’s Tumor Foundation’s Clinical Care Advisory Board and the 2015 recipient of CTF Humanitarian Award. Dr. Rosser serves as the Los Angeles site Principal Investigator for the U.S. Army Department of Defense NF Consortium which coordinates multi-center clinical trials for individuals with NF1 and Schwannomatosis. She is a member of the DOD NF Consortium Quality of Life and Neurocognitive Committees. She cares for many children and adults with both NF1 and Schwannomatosis. She is a collaborator on translational NF1 and Schwannomatosis research projects with researchers across the country.
Benjamin Housden
Benjamin Housden is an Associate Professor at the Living Systems Institute, University of Exeter, UK. His research focuses on NF1-associated plexiform neurofibromas, a major clinical complication of neurofibromatosis type 1.
Ben’s group uses a diverse and integrative set of experimental models, ranging from Drosophila and human cell systems to zebrafish, to study tumour biology and disease mechanisms. A central aim of his work is the use of genetic screening approaches to identify candidate drug targets that could inform new therapeutic strategies for tumour treatment.
Beyond plexiform neurofibromas, his research also explores how zebrafish models can be used to investigate the broader spectrum of NF1-associated symptoms and underlying disease mechanisms. More recently, his group has initiated a project aimed at developing preventative therapies to halt the progression of plexiform neurofibromas into malignant peripheral nerve sheath tumours.
In addition to his academic research, Ben serves as the Industry Partnerships Manager for the UK Human Functional Genomics Initiative, a UKRI-funded programme that brings together functional genomics researchers across academia and industry to accelerate the development of new therapeutics across a wide range of disease areas.
Chelsea Kotch
Children's Hospital of Philadelphia
Dr. Chelsea Kotch is a Pediatric Neuro-Oncologist at the Children’s Hospital of Philadelphia and an Assistant Professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine who specializes in the care of children with neurofibromatosis and central nervous system tumors. She received her MD from Tufts University School of Medicine and completed her pediatrics residency at Johns Hopkins Hospital and pediatric hematology/oncology and neuro-oncology fellowships at the Children’s Hospital of Philadelphia. Her additional training includes a Master of Science in Clinical Epidemiology and Biostatistics from the University of Pennsylvania. Dr. Kotch is a Francis S. Collins Scholar in NF Clinical and Translational Research with a clinical research program focused on improving outcomes for individuals with NF1-associated tumors through advanced epidemiologic methods and interventional clinical trials.
Florent Elefteriou
Baylor College of Medicine https://www.bcm.edu/people-search/florent-elefteriou-20948
Dr. Florent Elefteriou is a skeletal biologist whose career has centered on how bone develops, remodels, repairs, and communicates with other tissues in health and disease. He is a Professor of Orthopedic Surgery and of Molecular and Human Genetics at Baylor College of Medicine, and serves as Associate Director of the Center for Skeletal Medicine and Biology.
His laboratory has built a broad research program spanning skeletal development, mineralization, aging, repair, cancer metastasis to bone, neuroskeletal biology, and chondrocyte biology. A major focus of his scientific career has been on the orthopedic manifestations of NF1 such as fracture non-union, tibial pseudarthrosis and dystrophic scoliosis. His work helped define NF1 skeletal disease as a disorder intrinsic to bone and cartilage, rather than only a secondary consequence of systemic disease. Using genetic mouse models and pharmacologic approaches, his group clarified how loss of neurofibromin disrupts endochondral bone formation, remodeling, repair, and mineralization. He also contributed important mechanistic studies showing that neurofibromin restrains Ras/ERK-dependent FGFR signaling during skeletal development. Among his most influential NF1-related advances was the demonstration that Asfotase-alfa could improve bone growth, mineralization, and strength in mouse models of NF1, providing a clear translational therapeutic concept. Beyond NF1, Dr. Elefteriou is widely recognized for helping establish neuroskeletal biology, showing that the skeleton is under direct neural control and identifying pathways through which the autonomic nervous system regulates bone mass and remodeling. His research also expanded into cancer-bone interactions, including neural regulation of breast cancer skeletal colonization, and into cartilage biology through work on TonEBP/Nfat5 in chondrocytes.
Levi Kaster
Washington University School of Medicine
Levi is a 2nd year PhD candidate in Biomedical Informatics and Data Science at Washington University in St. Louis, where he applies artificial intelligence and natural language processing tools to derive insights from multimodal electronic health record (EHR) data in pursuit of improved clinical care and outcomes. He has worked on numerous projects related to NF1, including developing large language model (LLM) and rule-based pipelines to extract NF1-related phenotypes from clinical notes, and building machine learning models that leverage EHR data to perform early warning prediction of optic pathway gliomas.
Meena Upadhyaya
Cardiff University
Prof Dame Meena Upadhyaya DBE OBE PhD FRCPath FLSW is an Emeritus Professor in the Division of Cancer and Genetics at Cardiff University and an Honorary Professor of Practice at the University of Wales Trinity Saint David. She is a Fellow and Council Member of both the Royal College of Pathologists and the Learned Society of Wales.
Her research career has focused on a range of genetic disorders, with particular contributions to the understanding of neurofibromatosis type I (NF1) and facioscapulohumeral muscular dystrophy (FSHD). She has published over 200 papers and edited six books. Her achievements have been recognised through numerous honours, including the European Theodor Schwann Award for outstanding contributions to NF1 research (2013), an OBE (2016), the St David Award (2017), and a DBE (2026).
Meena served as a Non-Executive Director on the Civil Service Board of the Welsh Government from 2020 to 2024. She is the founder and Chair of the Ethnic Minority Welsh Women Achievement Association (EMWWAA) and the Ethnic Minority Women in Welsh Healthcare (EMWWH). She is also a trustee of Nerve Tumours UK, the European NF Group, Race Equality First, and Race Council Cymru. In addition, she is Co-chair of the Welsh Ethnic Minority Professors Initiative (WEMPI) and continues to organise the Festival of Communities in partnership with the Welsh Government.
Oliver Hanemann
University of Plymouth
Oliver has been advancing NF2 related schwannomatosis research for decades focussing on meningioma and schwannoma.
He built the Brain tumour centre of excellence at the University of Plymouth and was instrumental in University Hospitals Plymouth being awarded Tessa Jowell Centre of Excellence.
Soniya Chatterjee
Brain Tumor Institute
Soniya Chatterjee, PhD, is a Staff Scientist at Children’s National Hospital, where she
develops human iPSC-derived cerebral organoid models to study neurofibromatosis type 1
(NF1)–associated tumor biology and neural microenvironmental interactions. Her work
focuses on how the neural organoid environment influences glioma progression, with
particular emphasis on cytokine-mediated signaling.
With over a decade of experience in molecular and cancer biology, her expertise includes
CRISPR/Cas9 genome editing, stem cell engineering, and advanced imaging and functional
assays. Previously, Dr. Chatterjee was a Visiting Fellow at the National Cancer Institute
(NIH), where she studied telomere biology and genome stability in cancer and stem cell
systems.
Her research aims to build and apply reproducible preclinical in vitro platforms to better
understand tumor–organoid interactions and support the development of more effective
therapeutic strategies.
Centennial DE
Session Co-Chairs:
Corina Anastasaki, PhD, Washington University in St. Louis
Benjamin Housden, PhD, University of Exeter
Liyam Laraba, PhD, University of Plymouth
10:00 AM - 12:40 PM - NF1
10:00 AM - 10:25 AM: Invited Talk: Clinical Care of the NF1 Skeleton: Recognition, Surveillance and Management
Laura Klesse, MD, PhD, UT Southwestern Medical Center
10:25 AM - 10:50 AM: Invited Talk: Treating the NF1 Skeleton: Promise, Mechanisms, and Translational Barriers
Florent Elefteriou, PhD, Baylor College of Medicine
10:50 AM - 11:05 AM: Invited Talk: From Bench to Bedside: Bridging the Clinical Data Gap in NF1-Glioma
Chelsea Kotch, MD, Children’s Hospital of Philadelphia
11:05 AM - 11:20 AM - Coffee Break
11:20 AM - 11:35 AM: Platform Talk: Multimodal Early Warning Prediction of Optic Pathway Glioma in NF1 Utilizing Large Language Models (LLMs)
Levi Kaster, BS, Washington University School of Medicine
11:35 AM - 11:50 AM: Platform Talk: An NF1-Deficient Brain Organoid Microenvironment Enhances Glioma Progression via Cytokines
Soniya Chatterjee, PhD, Brain Tumor Institute
11:50 AM - 12:05 PM: Platform Talk: Evaluating Cell Painting and image-based profiling strategies NF1 patient-derived 3D organoids
Michael Lippincott, University of Colorado Anschutz Medical Campus
12:05 PM - 12:25 PM: Panel Discussion: NF1 non-plexiform/MPNST manifestations
Moderator:
David Largaespada, PhD, University of Minnesota
Panelists:
Rosalie Ferner, MD, Guys and St Thomas' Hospital NHS Trust
Corina Anastasaki, PhD, Washington University in St. Louis
Matthias Karajannis, MD, MS, Memorial Sloan Kettering
30 June, 2026 12:25 pm
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01:25 pm
Lunch
Centennial Foyer
Box lunches are available for purchase daily, Saturday through Tuesday. To save time and avoid on-site concession lines, please purchase your box lunch(es) during registration.
30 June, 2026 01:25 pm
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03:35 pm
NextGen Translational Pipelines: From Models to Clinical Impact, Afternoon Session
Matthias Karajannis
Memorial Sloan Kettering
Dr. Matthias Karajannis has been serving as the Chief of the Pediatric Neuro-Oncology Service at Memorial Sloan Kettering Cancer Center since 2017. He is a Member of the Memorial Hospital faculty and Professor of Pediatrics at Weill Cornell Medical College in New York. Previously, he directed the NF Clinical Research Program and the Pediatric Hematology/Oncology Fellowship Training Program at NYU Langone Medical Center. He received his MD from the Free University Berlin, Germany, completed residency training at Duke University Medical Center, and fellowships in pediatric hematology/oncology, as well as pediatric neuro-oncology, at Memorial Sloan Kettering Cancer Center. Dr. Karajannis treats children and young adults with tumors of the nervous system, and has a special interest in caring for patients with NF2-SWN. His research aims at developing novel diagnostic tools and molecular targeted therapies for patients with nervous system tumors, including NF2-SWN patients, through translational research and clinical trials. Dr. Karajannis serves as principal investigator and study chair for cooperative group clinical trials with the Children’s Oncology Group (COG), Pediatric Brain Tumor Consortium (PBTC) and NF Clinical Trials Consortium (NFCTC), where he chairs a recently completed phase 2 study for patients with NF2-SWN and progressive vestibular schwannoma.
Ayyapa Raja
California Institute of Technology
NF2 is a member of the ERM (ezrin, radixin, moesin) family of cell adhesion proteins and
codes for the protein Merlin, which acts as a tumor suppressor. Merlin functions as a
membrane-cytoskeleton linker that inhibits cellular proliferation via contact-dependent
regulation of various signaling pathways, including WNT/β-catenin, Notch, Ras, Rac/Rho,
TGF-β, Hippo, and receptor tyrosine kinases. Germline/Somatic mutations in the NF2 gene
lead to a genetic disorder, Neurofibromatosis type 2, characterized by Vestibular
Schwannomas and Meningiomas. In this talk, I will discuss generating a conditional
zebrafish model of neurofibromatosis established by an inducible genetic knockout of nf2,
the zebrafish homolog of human NF2. Gene expression analysis via Hybridization Chain
Reaction (HCR) reveals expression of nf2 in the neural crest and its derivatives, as well as in
the cranial mesenchyme. Induction of nf2 knockout at early stages leads to
hyperproliferation of larval Schwann cells and meningeal fibroblasts. Subsequently, in adult
zebrafish, nf2 knockout triggers the development of a spectrum of tumors, including
vestibular Schwannomas, spinal Schwannomas, meningiomas, and retinal hamartomas,
mirroring the tumor manifestations observed in patients with NF-2. Collectively, these
findings highlight the generation of a novel zebrafish model that mimics the complexities of
the human NF-2 disorder. Moreover, it holds promise for testing therapeutic agents or large-
scale chemical libraries for their ability to ameliorate NF-2 phenotypes. The accessibility,
ease of manipulation, availability of genetic tools, and ease of imaging promise to make this
an extremely useful model for further exploration of tumor ontogeny and for assessing
potential treatments.
Beomhee Lee
Asan Medical Center
BEOM HEE LEE (M.D., Ph.D.) is a Pediatrician and Medical Geneticist at Asan Medical Center, Seoul, Korea. Also serves as a Professor at University of Ulsan College of Medicine, Seoul, Korea. He participated in multiple clinical trials with focus studies on rare genetic disorders such as Neurofibromatosis, Lateralized overgrowth syndrome, Fatty acid oxidation disorder and Lysosomal storage disorders.
Corina Anastasaki
Washington University in St. Louis
Corina Anastasaki, obtained her PhD in Human Genetics and Developmental Biology in E. Elizabeth Patton’s lab (Edinburgh University, Scotland UK) where she developed zebrafish models of RASopathies. She continued her postdoctoral training at Washington University in St Louis with David H. Gutmann, where she established the first human biorepository of NF1 patient iPSC lines, and used them to uncover the primary role of the NF1 mutations in normal human (organoid) and mouse brain development as well as low-grade glioma (LGG) formation. She remained at Washington University’s NF Center first as an instructor and now an assistant professor to better characterize the function of central and peripheral nervous system neurons in initiating, maintaining and driving LGG and cutaneous neurofibroma growth (cancer neuroscience), leveraging her expertise in human iPSCs modeling, primary pediatric glioma cell generation, and preclinical humanized mouse LGG platform development. Her work focuses on furthering our understanding of molecular mechanisms driving brain (glioma) and nerve (neurofibroma) tumorigenesis and progression, in order to develop improved risk assessment and translational precision oncology tools, and her findings have already led to the identification of FDA-approved drugs that can be repurposed for the treatment of nervous system tumors.
Frank Buono
Yale School of Medicine
Frank Buono is a Research Scientist in the Department of Psychiatry and a secondary appointment in the Department of Orthopaedics and Rehabilitation at Yale School of Medicine. In addition, Dr. Buono is the co-director of the Neurocutaneous Clinic at Yale New Haven Hospital and the clinical director of research at Greenwich Hospital.
Dr. Buono’s research centers around digital technologies and innovations (e.g., mobile applications, web-based digital interventions, artificial intelligence) that facilitate treatments for individuals with rare diseases. Over the last ten years Dr. Buono has focused my research on the understanding of chronic pain in Neurofibromatosis centering on the interconnection of quality of life, as well as complimentary-alternative approaches for pain management. He has developed a customized mobile application for chronic pain management for adults with Neurofibromatosis type 1 (iCanCope-NF) which has shown success in reducing pain interference, while improving pain self-efficacy and pain acceptance. Additionally, Dr. Buono has created an AI based 3-D radiological software to accurately evaluate NF tumor growth which will soon have FDA approval.
Gareth Evans
University of Manchester UK
A Consultant at Manchester University Hospitals NHS Foundation Trust and The Christie NHS Foundation Trust, Professor Evans has established a national and international reputation in clinical and research aspects of cancer genetics, particularly in neurofibromatosis, schwannomatosis and breast cancer. Professor Evans is a clinician scientist and is an emeritus chair of medical genetics and cancer epidemiology at the University of Manchester. He has published 1180 peer reviewed research publications, 375 as first or senior author as well as over 160 reviews, letters and chapters. In the last eight years he has raised over £75 million in grants for multicentre and local studies. He was Chief Investigator on two NIHR program grants on breast cancer risk prediction and also has an NIHR RfPB grant as CI (2011). He has led a successful bid for a Nationally funded NF2 service (£7.5 million pa) that started in 2010 and is involved in the national complex NF1 service. He was the cancer prevention early detection theme leader on the NIHR Manchester Biomedical Research Centre. Professor Evans was also lead clinician on the NICE Familial Breast Cancer Guideline Group. H has a Web of Science h index of 156 and google scholar of 197
Liyam Laraba
University of Plymouth
I am passionate about generating innovative preclinical research that can be translated into clinical trials, to provide real benefit for NF2-related schwannomatosis patients. I have nine years’ experience working in the field of neuro-oncology, firstly as a PhD student, then as a Children’s Tumor Foundation Young Investigator awardee and currently as a tenure-track Senior Research Fellow. My institution is the University of Plymouth in the UK, we are a Brain Tumour Research Centre of Excellence specialising in low grade tumours. Our translational pipeline ranges from our large primary tumour biobank to 3D models, and now, the development and characterisation of a humanised mouse model of meningioma to facilitate immunotherapy drug testing.
Jeremie Vitte
University of California Los Angeles
Jeremie Vitte, PhD is a Project Scientist in the Department of Head and Neck Surgery at the David Geffen School of Medicine of the University of California Los Angeles (UCLA). Dr. Vitte received his PhD in Cellular and Molecular Biology in France and worked in the field of neuromuscular (Spinal Muscular Atrophy) and neurodegenerative (Parkinson’s) diseases. Dr. Vitte’s current research in the laboratory of Dr. Giovannini is focused on understanding the molecular and cellular mechanisms underlying tumor development in NF2-related and other types of schwannomatosis, by developing new genetically engineered mouse models as well as performing pre-clinical research and drug testing for these and related tumor predisposition syndromes.
Centennial DE
Session Co-Chairs:
Rosalie Ferner, MD, Guys and St Thomas' Hospital NHS Trust
Oliver Hanemann, MD, FRCP, University of Plymouth
Matthias Karajannis, MD, Memorial Sloan Kettering
1:25 PM – 3:35 PM: NF2-SWN
1:25 PM - 1:50 PM: Invited Talk: NF2-related schwannomatosis: From clinical trials to clinical application of drugs
Gareth Evans, MD, University of Manchester UK
1:50 PM - 2:15 PM: Invited Talk: Objective, Consistent, and Longitudinal MRI Measurement in NF2-Related Schwannomatosis: Why AI-Enabled 3D Volumetrics Matter for Care and Lived Experience
Frank Buono, PhD, Yale School of Medicine
2:15 PM - 2:40 PM: Invited Talk: Modeling Neurofibromatosis type 2 in zebrafish
Ayyapa Raja, PhD, California Institute of Technology
2:40 PM - 2:55 PM: Platform Talk: Single-cell RNA-sequencing in tumor-bearing humanized mice recapitulates human meningioma and refines preclinical drug discovery
Liyam Laraba, PhD, University of Plymouth
2:55 PM - 3:10 PM: Platform Talk: Clinical Evaluation of PRG-N-01 in NF2-Related Schwannomatosis: Results from a Phase 1 Study Beomhee Lee, PhD, Asan Medical Center
3:10 PM - 3:30 PM: Panel Discussion: NF2-SWN
Moderator:
Matthias Karajannis, MD, MS, Memorial Sloan Kettering
Panelists:
Frank Buono, PhD, Yale School of Medicine
Jeremie Vitte, PhD, University of California Los Angeles
Ayyapa Raja, PhD, California Institute of Technology
3:30 PM - 3:35 PM: Closing Remarks
30 June, 2026 03:35 pm
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03:45 pm
Closing Remarks & Adjournment
Centennial DE
